Q&A

Penn's Kiran Musunuru talks to us about the technology that has been both praised and criticized for its ability to alter human DNA and potentially cure disease.

Kiran Musunuru is an associate professor of medicine in genetics in the Perelman School of Medicine at the University of Pennsylvania. / Courtesy

CRISPR, the technology being used to edit genes in humans, remains polarizing. On one end, detractors argue that using the technology for certain purposes, like performing gene editing on embryos, is not only dangerous but unethical. On the other end, proponents say CRISPR has the potential to revolutionize human health, and early data shows they might be right. Despite a medical community that is still split on the issue, researchers in the U.S. are kicking tests of the technology into high gear. Several clinical trials have launched in the U.S. testing CRISPRs ability to treat various diseases.

NextHealth PHL spoke with Kiran Musunuru, an associate professor of medicine in genetics at the Perelman School of Medicine at the University of Pennsylvania about the true potential of CRISPR technology and how we can expect it to evolve in the future.

NextHealth PHL: What exactly is CRISPR?Musunru: CRISPR is sort of a catch-all term that covers a variety of technologies. If youre saying CRISPR, youre referring to a broad set of tools that may do it in different ways but are all intended to do a form of gene editing or genome editing.

How do basic CRISPR technologies work?The simplest form of CRISPR, what I call version 1.0, is the original standard CRISPR that most laboratories and companies interested in developing new therapies use. It is a two-component system. There is a protein and an RNA molecule thats about 100 bases in length. The protein and the RNA molecule come together to create what well call a molecular machine and the purpose of this molecular machine is to scan across any DNA molecule it encounters. So if you put the CRISPR-Cas9 into the nucleus of a human cell, this molecular machine will scan the entire genome.

The machine has two key functions built into it; the first is a GPS function. When you change the first 20 bases in a DNA length (the first 20 bases is basically the address) to whatever address you want, the GPS function makes the machine go through the entire genome and find the sequence that matches the address. The second function of this machine is to protect the genome, like a search-and-destroy function. You put in the address, it goes to that matching place in the genome and then it makes a cut in the DNA.

Cutting the DNA is actually a bad thing but the cells have ways to try to fix that break, and the actual editing is a result of the cell trying to fix that break in the DNA, not from CRISPR itself, interestingly enough.

How does CRISPR turn a break in someones DNA into a good thing?There are a few ways this can happen. The safest thing you can do is to break a gene or turn off a gene. The metaphor I like to use is to think of the whole genome as a book, and each chromosome in the genome is a chapter in the book, and each gene is a paragraph in the chapter. Together, it all has a meaning. But lets say you had to turn off a gene, the equivalent of making that break in the DNA would be like tearing the page through that paragraph. So, the simplest thing the cell can do and will try to do is to simply tape that tear back up. But as you can imagine, sometimes you tape it back up and its fine, the paragraph is still legible and the meaning is still there, and it eventually heals and functions like it did before. But in this case, thats actually not what you want. The outcome that you want with CRISPR is that you actually want to turn off the gene, not to rip it and make it the way it was before.

What has to happen is when you make the tear, the tear is so rough, you get those jagged edges and you try to tape it up but it doesnt quite fit, the letters dont quite match up. You tape it up as best as you can but its illegible, some letters are lost, and the meaning of the paragraph is lost. Thats exactly what happens with gene editing, the cell tries to repair that break in the DNA, doesnt get it quite right, and loses some bases and that messes up the gene and turns it off.

However, in this scenario, you cant really control what happens. All you can hope for is that that tear you make is going to mess up the gene and thats okay if all youre trying to do is turn it off. Most of the trials underway now are about turning off the gene, and theyre all taking advantage of the fact that its relatively easy to mess up genes and turn off genes. Just like tearing a page its crude, but its effective.

Theres CRISPR 1.0, this first generation of the technology thats not very precise and is a bit arduous. What are the newest forms of CRISPR and how are they better than earlier versions of the technology? There is a newer form of the technology called base editing that keeps the GPS function intact but removes the cutting function. In place of the cutting function, it attaches another machine onto CRISPR and makes chemical modifications in certain areas. This version of CRISPR is more like a search and replace. CRISPR provides the search but then another machine attached to it is doing the replacing. With base editing you can make more precise changes, but only rarely will it make exactly the type of change you want.

The latest form of CRISPR is called prime editing, and we still dont have a good sense of how well it works because its so new. Whats tantalizing is that it looks like it can turn CRISPR into a precise word processor or an eraser that allows you to erase a letter and put in a new letter. CRISPR is very much a wave of technology, and as it gets better, its going to allow us to do more and more powerful things.

There are some extreme ideas about what CRISPR can do. Some believe scientists can use the technology to alter hair or eye color or give patients superhuman athletic or intellectual abilities. Is any of this possible with CRISPR?It depends on what traits youre talking about changing. Since eye color and hair color are controlled by single genes, you could possibly make a single gene change with CRISPR. The problem is, how do you get CRISPR to go where it needs to go to change your hair or eye color? How do you get it into all your hair follicles or through all the cells in your eye? It might be a simpler change to make, but it might not be easy to do in a live adult. Scientists have now edited human embryos, resulting in live-born people. Theres been a lot of ethical debate about whether thats a good thing. If you want to change something like hair color in a single cell embryo made through in-vitro fertilization, thats a bit different and might not be as difficult.

There are some very complicated things, like intelligence or athletic ability, that are not going to be easy to change. Youd probably have to change hundreds of genes, and thats not going to happen anytime soon. With CRISPR as it is now, maybe you can change one gene; maybe if you really work at it you can change two genes, but hundreds of genes? Youre not going to be able to do that with CRISPR anytime soon.

What has CRISPR been used to treat so far and what could it be used for in the future?There are multiple trials underway to treat rare liver disorders. More recently CRISPR has been used in clinical trials at Penn where at least three patients have been dosed using CAR T immunotherapy. In this case, theyre trying to make patients cells more effective at fighting cancer. But again, that editing is being done outside the body.

There are some things that seem like they would be difficult to treat, but if its the right type of disease and you can get CRISPR to where you need it to go, it might work. One example is in sickle cell disease. The cells that you need to fix in sickle cell disease are in the bone marrow. Fortunately, bone marrow is relatively straight forward to work with. You take the cells out and edit them with some form of CRISPR outside of the body and then put them back in.

Something like cystic fibrosis would be much harder because it affects the entire surface of potentially multiple organs inside the body. Its much harder to deliver CRISPR to all of those places in the body.

There are two other clinical trials that have started in the U.S. One is from a company called CRISPR Therapeutics to treat sickle cell disease and similar blood disorders. Theres another trial underway to treat a genetic form of blindness and this editing would actually happen inside the body.

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Q&A: Everything You Need to Know About the Future of CRISPR-Cas9 - Philadelphia magazine

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Professor Glenn Cohen is a Professor of Law at Harvard Law School. He is also the director of Harvard Law School's Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics, and one of the world's leading experts on the intersections of bioethics and the law. Cohen's current projects relate to big data, health information technologies, reproduction/ reproductive technology, research ethics, organ rationing in law and medicine, health policy, FDA law, translation medicine, and medical tourism. The utilization of CRISPR technology as a gene editing tool has spurred significant debate across the globe. In this interview, we gain insight of Cohen's perspectives on the "CRISPR revolution" and learn about the basic ethical issues surrounding the manipulation of the genome for enhancement.

Molly Campbell (MC): You are one of the leading experts on the intersection of bioethics and the law. Please can you tell us more about this field and the types of cases it addresses?Glenn Cohen (GC): Wherever law, medicine, and ethics intersect, thats where the field and I are. Whether it is the ethics of research, reproductive technologies, genetics, end of life decision-making, mental health, neuroscience, rationing, AI, clinical practice, etc. It is a robust and very exciting field.

MC: Currently, what restrictions apply to the use of CRISPR technology in different cell types and organisms? What applications are scientists not allowed to use CRISPR or other gene-editing technologies for? GC: In lay terms, in the United States an appropriations rider prohibits FDA from considering the use of germline gene editing in human beings. Thus, it is not possible to do a clinical trial or the like of this. Many (perhaps all, it is not clear everywhere) other countries across the world also prohibit in one way or another, but not all regulatory regimes may be as effective.

MC: The work of Jiankui He arguably startled the scientific community. In your opinion, do you think the publication of He's work prompted authorities to address regulating CRISPR technology? Or was there already a conversation taking place?GC: There was very robust conversation long before Dr. Hes terrible (and in my view completely unethical) experiments. For example, this report from the National Academies. While CRISPR is relatively new in terms of technology, in fact bioethicists have been talking about the basic issues surrounding manipulating the genome for enhancement for at least 40 years if not longer.

MC: There are concerns that the CRISPR tool could be used for enhancement purposes. In recent opinion article you say, "Anyone who has a position on enhancement has not thought deeply enough on the question." Please can you expand on what you mean by this?

GC: My claim is that enhancement is not a single monolithic thing, so it is hard to have a single position on it. Some enhancements would be wonderful and perhaps the state should subsidize them. Others would be terrible and perhaps the state should prohibit. Only when we think about it with some specificity can we know what we think the answer should be. In the article you mention I draw the following distinctions, for example, though others are possible:

1. Biological vs. Non-Biological Enhancement

a. Genetic enhancements vs. non-genetic biological enhancements

2. Choosing for Ourselves vs. Choosing for Others Who Cannot Choose for Themselvesa. Enhancing after birth vs. enhancing before birthi. Enhancing by selection vs. enhancing by manipulation of already fertilized embryos or implanted fetuses

3. Enhancements Compatible with Expanding Life Plans vs. Enhancements That Will Limit Options

4. Reversible vs. Irreversible Enhancement

5. Some would distinguish enhancement from treatment (though others are skeptical about this distinction)a. Enhancements to the upper bounds of what people already have vs. enhancements that add beyond human nature as it now stands

6. Enhancements for Absolute vs. Positional Goods

MC: A novel community of gene-editing "biohackers" has emerged in the rise of CRISPR technology. What are your opinions of biohackers conducting gene-editing experiments from their homes, from a legal and ethical perspective?GC:I think the community is very interesting. I am a huge fan of open science and the building of intellectual communities. I think the key question is whether/when the work undertaken by this community could pose significant externalities for others. Thats probably where I would start to get concerned.

MC: How do we approach implementing a global legal and ethical framework for using gene-editing technologies? What progress has been made thus far?GC: The WHO has chartered an advisory committee which has recommended a registry of all those doing gene editing work and has advised that it is irresponsible at this time for anyone to proceed with clinical applications of human germline genome editing." I think the existence of this committee (alongside the NASEM, Nuffield Council) and others working on these issues is a great step.

My own view is that we ought to be looking for a responsible translational pathway that might allow some clinical work to be reviewed and approved by regulators like the FDA in the future, but certainly there is nothing there yet. The international aspect makes this very, very difficult. Some have suggested we ought to go for an international treaty, like what we have on landmines and chemical weapons but also recognition of adoption, while others think this is infeasible.

MC: What challenges exist when looking to create laws surrounding a novel scientific technology?GC: There are quite a few. The first is uncertainty whenever you move to first-in-humans, whatever pre-clinical work you have done, there is always open questions. The same was true with IVF. The second is the politicization of science and the reduction of difficult and nuanced questions to talking points. The third is deep philosophical disagreement on some key points (for example, some take quite literally the idea of "man created in Gods image" and view altering the human genome as a rejection of that. If thats what someone believes for religious reasons then it is very hard to talk about these issues at a more policy level). Fourth, is the importance but difficult of public engagement. The UK in its public consultation on mitochondrial replacement therapy (that ultimately paved the way for permitting that technology to be used in a limited way) was a very good recent model, but quite difficult and expensive. Moreover, some felt it didnt go far enough in the direction of deliberative democracy. The hope is we will see more such initiatives for gene editing and other novel technologies.

Professor Glenn Cohen, Haravard Law School, was speaking with Molly Campbell, Science Writer, Technology Networks.

Catch up on the previous instalment of Technolology Networks Explores the CRISPR Revolution, an interview with Professor George Church, here.

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Technology Networks Explores the CRISPR Revolution: An Interview With Professor Glenn Cohen, World-leading Expert on Bioethics - Technology Networks

Recommendation and review posted by Bethany Smith

ByKristin Hanson

This article was originally published on Nov. 8 on giving.jhu.edu

Between 2001 and the beginning of 2018, more than 1,500 U.S. military service members lost limbs in the line of duty. Although technology has improved the prosthetic devices these people can use, a stubborn obstacle remains: the fragility of human skin.

"Skin was never meant to hold this kind of pressure," says Lee Childers, the senior scientist for the Extremity Trauma and Amputation Center of Excellence at Brooke Army Medical Center in San Antonio, Texas.

"Think about it like a blister on your foot. It's painful, but you can still get by," he continues. "In an amputation, it's a blister on your residual limb. You can't use your prosthesis until the blister is completely healed. If it's your leg [that is affected], you can't walk for two or three weeks. Think about how that would impact your life."

What if there were a way to make the skin at an amputation site tougher, like the palm of your hand or the sole of your foot? Luis Garza, an associate professor of dermatology at Johns Hopkins and leader of the Veteran Amputee Skin Regeneration Program, is developing a cell therapy that could enable prosthetics wearers to use their devices longer.

"This is an example of personalized medicine," Garza says. "We're taking each person's own cells, growing them up, and inserting them back in."

Garza's postdoctoral research focused on skin stem cells. In 2009, he and his department chair, Sewon Kang, began having conversations about how that work could help the increasing numbers of veterans coming back from war with amputations. Garza and his team received grants from the U.S. Department of Defense, National Institutes of Health, and Maryland Stem Cell Fund that have moved the program forward in the past decade.

Garza's team spent the summer of 2019 testing "normal" subjectsthose without amputationsto perfect the procedure, including the dose, content, method, and frequency of the injections. During one appointment, members of Garza's team took biopsies of skin from a subject's scalp and sole. The cells went to a lab where they were grown under an FDA-approved protocol and passed through quality control tests.

In a second appointment, subjects completed a questionnaire and underwent baseline measurements of their skin's thickness and strength. Garza's team then injected a site on the subjects' skin with the stem cells grown from their cells in the lab.

Image caption: Luis Garza, associate professor of dermatology at Johns Hopkins, leads the Veteran Amputee Skin Regeneration Program.

"We're hoping that these stem cell populations will engraft in the new skin," Garza says.

The subjects returned to Hopkins several months later to go through the questionnaire and measurements once more, and Garza's team documented changes.

Confident in the results they gleaned from the normal subjects, Garza's team enrolled its first subject with an amputation in August. Moving from the normal population to the amputation-affected population quickly unearthed some aspects of the therapy Garza didn't anticipate.

"When we talked with him, he said 'I don't want to mess with my one remaining footdo you have to take skin from there?' And we said, 'Actually, no, we could do your palm,'" Garza says.

His team then tested the biopsy and growth of palm cells from subjects in the normal population. "We're moving away from having our product informed purely by biology to letting our therapy development be shaped by the user."

Although federal grants have supported much of the program's progress, private philanthropy has played a role, too. Corporations like Northrop Grumman, foundations like the Alliance for Veteran Support, and grateful patients with and without ties to the armed forces have contributed nearly $300,000. Those gifts have enabled the program to persevere through gaps between federal grants.

Private funds will be increasingly important as the project enters its next phase: extension to military medical centers around the country. Garza's team must prove that the safeguards to protect cells on their round-trip voyage from a test site to Hopkins are effective. They also must secure approval by local institutional review boards for clinical studies.

"Soldiers are used to getting orders, but you can't order someone to be part of a [medical] study," Garza says. "There are hard medical ethics questions around how to make this open to them but ensure they don't feel obligated. We've been working on that for a year, and we probably have another six months or so to go."

Childers stands ready for whenever the program's extension is a go. He will lead the study at Brooke Army Medical Center and feels motivated by the prospect of helping many of the veterans he works with every day.

"We do everything we can to serve those who serve us. This can enable people to return to duty and be redeployed if they choose," he says. "This is game-changing technology that will have an impact for our service members, but also others who live with amputation."

That population includes the hundreds of thousands of Americans who've undergone amputations for complications of diabetes, who must use a wheelchair, or who wear ankle or foot orthoses for help with walking, among others.

"Having the ability to transform skin anywhere you want to target on the body will have gigantic implications across the entire spectrum of our society in many ways," Childers says.

There's a lot of work to be done before such benefits reach the public, Garza cautions. With continued support from donors and the military community, though, he's optimistic about the program's future.

"The challenges are pretty big, but I think within five years, it could happen," he says. "That's the hope."

Disclaimer: The view(s) expressed herein are those of the author(s) and do not reflect the official policy or position of the Brooke Army Medical Center, the U.S. Army Medical Department, the U.S. Army Office of the Surgeon General, the Department of the Army, the Department of the Air Force and Department of Defense or the U.S. Government.

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Serving those who serve - The Hub at Johns Hopkins

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Even though your skin goes through unlimited wear and tear, it is able to repair itself. How does it undertake these regrowth factory activities? There are reservoirs of stem cells within supportive micro-environments, also called niches, throughout the skin. They can keep a tight leash on this repairing process. Too much tissue can lead to complications such as cancer, even though too less may enhance ageing.

Can stem cells direct other stem cells to reform into new skin when they reshape their niches? A study published in Science, led by Prof. Elaine Fuchs actually shows that stem cells have an effect on tissue regeneration. It marks out a molecular coordination tool that can be leveraged by stem cells to convey signals across niches.

The scientists wrote in the abstract of their article, published in Science: "Tissues rely on stem cells (SCs) for homeostasis and wound-repair. SCs reside in specialized microenvironments (niches) whose complexities and roles in orchestrating tissue growth are still unfolding. Here, we identify lymphatic capillaries as critical SC-niche components. In skin, lymphatics form intimate networks around hair follicle (HF) SCs."

Hence, another component of the niche that was revealed was the lymphatic capillaries, specialized types of vessels. They transport immune cells and also drain out excess fluids and toxins from tissues. As the capillaries integrate into a close network around the stem cell niche inside every hair follicle, all the niches get interconnected.

"By turning the skin completely transparent," says postdoctoral fellow Shiri Gur-Cohen, "we were able to reveal the complex architecture of this network of tubes." Researchers identified that the hair-follicle stem cells manage the behavior of lymphatic capillaries. They do this by secreting molecules acting as on-off switches for drainage. They enabled them to monitor the compositions of fluids and cells around them and finally synchronize regeneration across the tissues.

"The involvement of the lymphatic system in this process is a new concept," says Fuchs, "and might potentially provide new therapeutic targets for lymph-related conditions such as wound-healing defects and hair loss." The scientists summarized their findings in Science: "When lymphatics are perturbed or the secretome switch is disrupted, HFs cycle precociously and tissue regeneration becomes asynchronous. In unearthing lymphatic capillaries as a critical SC-niche element, we have learned how SCs coordinate their activity across a tissue."

Hence, to those who haven't understood why they are losing hair in tufts, checking out the scientific experiments on your lymphatic systems can go a long way in figuring out how it can be regrown.

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Hair regrowth depends on lymphatic system, finds study giving hope to find way to cure baldness - International Business Times, Singapore Edition

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Since 2006, when Shinya Yamanaka, now the director of the Center for iPS Cell Research and Application at Kyoto University, discovered a method that could guide fully differentiated cells back to their pluripotent state, scientists have been using his recipe to produce induced pluripotent stem cells. The protocol relies on overexpressing the so-called Yamanaka factors, which are four transcription factors: Oct4, Sox2, Klf4, and cMyc (OSKM). While the technique reliably creates iPS cells, it can cause unintended effects, some of which can lead to cells to become cancerous. So researchers have worked to adjust the cocktail and understand the function of each factor.

No one had succeeded in creating iPS cells without forcing the overexpression of Oct4. It was thought that this was the most crucial factor of the four. At least until now.

If this works in adult human cells, it will be a huge advantage for the clinical applications of iPS cells.

Shinya Yamanaka, Kyoto University

Four years ago, Sergiy Velychko, a graduate student at the Max Planck Institute for Molecular Biomedicine in Hans Schlers lab, and his team were studying the role of Oct4 in creating iPS cells from mouse embryonic fibroblasts. He used vectors to introduce various mutations of the gene coding for Oct4 to the cells he was studying, along with a negative controlone that didnt deliver any Oct4. He was shocked to discover that even using his negative control, he was able to generate iPS cells.

Velychkos experiment was suggesting that it is possible to develop iPS cells with only SKM.

We just wanted to publish this observation, Velychko tells The Scientist, but he knew hed need to replicate it first because reviewers wouldnt believe it.

He and his colleagues, including Guangming Wu, a senior scientist in the lab, repeated the experiment several times, engineering vectors with different combinations of the four factors. SKMthe combination that didnt include Oct4was able to induce pluripotency in the cells with about 30 percent of the efficiency of OSKM, but the cells were of higher quality, meaning that the researchers didnt see evidence of common off-target epigenetic effects. They reported their results yesterday (November 7) in Cell Stem Cell.

Efficiency is not important. Efficiency means how many colonies do you get, explains Yossi Buganim, a stem cell researcher at the Hebrew University of Jerusalem, who was not involved in the study. If the colony is of low quality, the chances that eventually the differentiated cells will become cancerous is very high.

Finally, the team employed the ultimate test, the tetraploid complementation assay, in which iPS cells are aggregated with early embryos that otherwise would not have been able to form a fully functional embryo on their own. These embryos grew into mouse pups, meaning that the iPS cells the team created were capable of maturing into every type of cell in the animal.

Whats more is they found that the SKM iPS cells could develop into normal mouse pups 20 times more often than the OSKM iPS cells, suggesting that the pluripotency of iPS cells can be greatly improved by omitting Oct4 from the reprogramming factor cocktail.

The results will need to be verified in human cells, Buganim cautions. His team has developed methods for creating iPSCs that worked well in mouse cells only to be completely ineffective in humans.

Yamanaka himself was enthusiastic about the results, telling The Scientist in an email that his team would definitely try the method in other cell types, especially adult human blood cells and skin fibroblasts. If this works in adult human cells, it will be a huge advantage for the clinical applications of iPS cells.

S.Velychkoet al.,Excluding Oct4 from Yamanaka cocktail unleashes the developmental potential of iPSCs,Cell Stem Cell,doi:10.1016/j.stem.2019.10.002,2019.

Emma Yasinski is a Florida-based freelance reporter. Follow her on Twitter@EmmaYas24.

Read this article:
Oct4, Considered Vital for Creating iPSCs, Actually Isnt Needed - The Scientist

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Hormonal changes can play havoc with our skin as we get older, especially in the perimenopause and menopause years.

These changes aren't the same for every woman, and they don't all begin at once.

During the perimenopause and menopause, the most notable hormonal change is a decline of oestrogen levels.

Oestrogen affects every tissue and organ of the human body, skin included, so the decline of oestrogen in perimenopause and menopause can cause the following symptoms:

Dermatologist and founder of Meder Beauty Science Dr Tiina Meder explained the best way to look after your skin during perimenopause and menopause is a "considered daily skincare routine".

"Using a gentle cleanser will help preserve the skins barrier function and prevent dryness and sensitivity," she said.

"Antioxidant-rich moisturisers, packed with prebiotics, will help keep skin hydrated and protected, simultaneously restoring and preserving the skins microbiome.

"Facial oils will also help to compensate lipid deficiency, while weekly exfoliation will help stimulate skin renewal.

Perimenopausal and menopausal skin is more sensitive to sun exposure.

"The maintenance of melanocytes the cells that manufacture the pigment melanin - is controlled by oestrogen," said Dr Meder.

"During the perimenopause and menopause, the number of melanocytes in your skin reduces andoestrogenlevels decline. As a result, less protective melanin is produced, making the skin appear lighter.

"As melanin helps protect the skin from the environmental damage and sun exposure, a decline in the production of melanin results in skin that is more prone to damage from sun exposure.

"As a consequence, it is very important to protect the skin regularly and correctly the second these hormonal changes appear."

When choosing skincare products thatll protect and repair skin during the perimenopause and menopause, Dr Meder recommends looking out for the following ingredients:

Moisturisers- hyaluronic acid, glycerine, carrageenan, chondrus crispus extract, gluconolactone and others.

Fatty acids and lipids- primrose, apricot, olive, macadamia, sweet almond, argan, borago, canola, meadowfoam, sunflowers, and sesame oil, as well as shea butter, squalane, cacao and, in some cases, coconut butter.

Prebiotics and probiotics- alpha-glucan oligosaccharide, inulin, and others, including some bacterial ferments and lysates (alteromonas filtrate, lactobacillus lysate, and saccharomyces).

Antioxidants- resveratrol, green tea, aloe barbadensis, rosemary and wild carrot extracts, vitamin E and C, and beta-carotene.

Remodelers- EGF (Epidermal Growth Factor) and others growth factors, plant stem cells, and peptides (Matrixyl-3000, Rigin, Syn-Tack and others).

Anti-inflammatories- centella asiatica, aloe barbadensis, green tea, calendula officinalis and chamomilla recutita extracts, panthenol, peptide skinasensyl, and albatrellus ovinus.

Microcirculation and capillary strengtheners- niacinamide (vitamin B3), caffeine, horse chestnut extract, and escin.

"Some ingredients - such as retinol or hydroxyl acids - can potentially increase the sensitivity of the skin, cause dryness, or increase ultraviolet sensitivity during the perimenopause and menopause," she went on.

"Sadly, many of these ingredients can actually help perimenopausal andmenopausal skin in many ways by improving the renewal process, lightening pigmentation, decreasing the appearance of wrinkles, and helping restore skin elasticity.

"Luckily, there are some great alternatives to these more aggressive ingredients. For example, retinol and retinol derivatives can be replaced with bakuchiol a natural ingredient that acts in a similar way to retinol - promoting the same benefits but with no side effects."

"Studies have found that HRT can provide several benefits to the skin. The reduction of oestrogen levels during the menopause has a detrimental effect on the skin, so it can be corrected, at least in part, through the early use of HRT in perimenopause.

"When HRT is introduced in the perimenopause period, skin dryness and sensitivity have been shown to be prevented. In addition, long-term use of HRT has been shown to restore the skins water-holding capacity and barrier function of the epidermis.

"Some studies have also found that HRT can control, in part, the dermal thickness and laxity, collagen content and density, as well as the skins mechanical properties and stress reaction."

Go here to read the rest:
Doctor explains what happens to your skin during the menopause and the best products to treat it - RSVP Live

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Global Stem Cell Therapy Market: Overview

Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.

Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.

Know the Growth Opportunities in Emerging Markets

Global Stem Cell Therapy Market: Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

The regional analysis covers:

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Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

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Stem Cell Therapy Market Poised to Expand at a Robust Pace Over 2025 - Tech Admirers

Recommendation and review posted by Bethany Smith

NEW YORK / ACCESSWIRE / November 11, 2019 / Kadmon Holdings, Inc. (KDMN) today announced positive topline results from the planned interim analysis of ROCKstar (KD025-213), the fully enrolled pivotal trial evaluating KD025 in patients with chronic graft-versus-host disease (cGVHD) who have received at least two prior lines of systemic therapy. The trial met the primary endpoint of Overall Response Rate (ORR) at the interim analysis, which was conducted as scheduled two months after completion of enrollment.

KD025 showed statistically significant ORRs of 64% with KD025 200 mg once daily (QD) (95% Confidence Interval (CI): 51%, 75%; p<0.0001) and 67% with KD025 200 mg twice daily (BID) (95% CI: 54%, 78%; p<0.0001). KD025 has been well tolerated and adverse events have been consistent with those expected in the patient population.

"We are extremely pleased with the outcomes of the interim analysis, which showed that KD025 has already greatly exceeded the threshold for success in this pivotal trial," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "We look forward to sharing these results with the FDA at a pre-NDA meeting, where we will also discuss the timing for a regulatory filing for KD025 in cGVHD, which we expect to occur in 2020, subject to FDA input."

"KD025 was shown to be a highly active and well-tolerated therapy across the spectrum of this complex, multi-organ disease," said Corey Cutler, MD, MPH, FRCPC, Associate Professor of Medicine, Harvard Medical School; Medical Director, Adult Stem Cell Transplantation Program, Dana-Farber Cancer Institute and a KD025-213 study investigator and Steering Committee member. "The response rates observed are particularly impressive since this study is being conducted in a real-world population with severe disease, supporting the potential role of KD025 in cGVHD patients who are in need of effective and well-tolerated therapies."

"It is highly encouraging to see the positive results from the pivotal trial are in line with those observed in the earlier Phase 2 study of KD025 in this difficult-to-treat disease," said Madan Jagasia, MD, Vanderbilt University, an investigator of the KD025-208 and KD025-213 studies and the KD025-213 Steering Committee chair. "These latest KD025 data continue to underscore the value that KD025 may offer to cGVHD patients."

KD025-213 is an ongoing open-label trial of KD025 in adults and adolescents with cGVHD who have received at least two prior lines of systemic therapy. Patients were randomized to receive KD025 200 mg QD or KD025 200 mg BID, enrolling 66 patients per arm. Statistical significance is achieved if the lower bound of the 95% CI of ORR exceeds 30%, which was achieved in both arms of the trial at the interim analysis.

While the ORR endpoint was met at the interim analysis, the primary analysis of the KD025-213 study will occur in the first quarter of 2020, six months after completion of enrollment. This analysis will include updated safety data and efficacy data, including ORRs and secondary endpoints, such as duration of response, changes in corticosteroid dose and changes in quality of life. Kadmon plans to submit results from the KD025-213 study for presentation at an upcoming scientific meeting.

Conference Call and Webcast

Kadmon will host a conference call and webcast on Monday, November 11, 2019, at 5:00 p.m., Eastern time, to discuss the topline results of the interim analysis of the KD025-213 study.

To participate in the conference call, please dial (866) 762-3021 (domestic) or (703) 925-2661 (international) and reference the conference ID: 6468498. The accompanying slides will be available for download on Kadmon's website beginning at 5:00 p.m. Eastern time.

To listen online via webcast, please visit: https://edge.media-server.com/mmc/p/9b9w8p38. The webcast will be archived and will be available at http://investors.kadmon.com/presentations-and-events.

About KD025

KD025 is a selective oral inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates inflammatory response. In addition to cGVHD, KD025 is being studied in an ongoing Phase 2 clinical trial in adults with diffuse cutaneous systemic sclerosis (KD025-209). KD025 was granted Breakthrough Therapy Designation and Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of patients with cGVHD who have received at least two prior lines of systemic therapy.

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About cGVHD

cGVHD is a common and often fatal complication following hematopoietic stem cell transplantation. In cGVHD, transplanted immune cells (graft) attack the patient's cells (host), leading to inflammation and fibrosis in multiple tissues, including skin, mouth, eye, joints, liver, lung, esophagus and gastrointestinal tract. Approximately 14,000 patients in the United States are currently living with cGVHD, and approximately 5,000 new patients are diagnosed with cGVHD per year.

About Kadmon

Kadmon is a biopharmaceutical company developing innovative products for significant unmet medical needs. Our product pipeline is focused on inflammatory and fibrotic diseases as well as immuno-oncology.

Forward Looking Statements

This press release contains forward-looking statements. Such statements may be preceded by the words "may," "will," "should," "expects," "plans," "anticipates," "could," "intends," "targets," "projects," "contemplates," "believes," "estimates," "predicts," "potential" or "continue" or the negative of these terms or other similar expressions. Forward-looking statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. We believe that these factors include, but are not limited to, (i) the initiation, timing, progress and results of our preclinical studies and clinical trials, including KD025-213, and our research and development programs; (ii) our ability to advance product candidates into, and successfully complete, clinical trials; (iii) our reliance on the success of our product candidates, including KD025; (iv) the timing or likelihood of regulatory filings and approvals, including in connection with KD025-213; (v) our ability to expand our sales and marketing capabilities; (vi) the commercialization of our product candidates, if approved; (vii) the pricing and reimbursement of our product candidates, if approved; (viii) the implementation of our business model, strategic plans for our business, product candidates and technology; (ix) the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates and technology; (x) our ability to operate our business without infringing the intellectual property rights and proprietary technology of third parties; (xi) costs associated with defending intellectual property infringement, product liability and other claims; (xii) regulatory developments in the United States, Europe and other jurisdictions; (xiii) estimates of our expenses, future revenues, capital requirements and our needs for additional financing; (xiv) the potential benefits of strategic collaboration agreements and our ability to enter into strategic arrangements; (xv) our ability to maintain and establish collaborations or obtain additional grant funding; (xvi) the rate and degree of market acceptance of our product candidates; (xvii) developments relating to our competitors and our industry, including competing therapies; (xviii) our ability to effectively manage our anticipated growth; (xix) our ability to attract and retain qualified employees and key personnel; (xx) our ability to achieve cost savings and other benefits from our efforts to streamline our operations and to not harm our business with such efforts; (xxi) the use of proceeds from our recent public offerings; (xxii) the potential benefits of any of our product candidates being granted orphan drug designation; (xxiii) the future trading price of the shares of our common stock and impact of securities analysts' reports on these prices; and/or (xxiv) other risks and uncertainties. More detailed information about Kadmon and the risk factors that may affect the realization of forward-looking statements is set forth in Kadmon's filings with the U.S. Securities and Exchange Commission (the "SEC"), including Kadmon's Annual Report on Form 10-K for the fiscal year ended December 31, 2018 and subsequent Quarterly Reports on Form 10-Q. Investors and security holders are urged to read these documents free of charge on the SEC's website at http://www.sec.gov. Kadmon assumes no obligation to publicly update or revise its forward-looking statements as a result of new information, future events or otherwise.

Contact Information

Ellen Cavaleri, Investor Relations646.490.2989ellen.cavaleri@kadmon.com

SOURCE: Kadmon Holdings, Inc.

View source version on accesswire.com: https://www.accesswire.com/566116/Kadmon-Announces-that-KD025-Met-Primary-Endpoint-at-Interim-Analysis-of-Pivotal-Trial-in-Chronic-Graft-Versus-Host-Disease

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Kadmon Announces that KD025 Met Primary Endpoint at Interim Analysis of Pivotal Trial in Chronic Graft-Versus-Host Disease - Yahoo Finance

Recommendation and review posted by Bethany Smith

SOMETIME NEXT year, if all goes to plan, a gay male couple in California will have a child. The child in question will have been conceived by in vitro fertilisation. In this case a group of eggs from a female donor are now being fertilised by sperm from both fathers (half from one, half from the other). Of the resulting embryos, the couple will choose one to be implanted in a surrogate mother. An uplifting tale of the times, then, but hardly a newsworthy event. Except that it is.

Where the story becomes newsworthy is around the word choose. For the parents, in conjunction with a firm called Genomic Prediction, will pick the lucky embryo based on a genetically estimated risk of disease. Such pre-implantation testing is already used in some places, in cases where there is a chance of parents passing on a condition, such as Tay-Sachs disease, that is caused by a single faulty gene. Genomic Prediction is, however, offering something more wide-ranging. It is screening embryos for almost 1m single-nucleotide polymorphisms (SNPs). These are places where individual genomes routinely differ from one another at the level of an individual genetic letter. Individual SNP differences between people rarely have much effect. But add them up and they can raise or lower by quite a lot the likelihood of someone suffering a particular disease. Generate several embryos and SNP-test them, then, and you can pick out those that you think will grow up to be the healthiest.

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Much fuss was made last year about a researcher in China, He Jiankui, who edited the genomes of two human embryos in order to try, he claimed, to make them immune to infection by HIV, the virus that causes AIDS. What Genomic Prediction proposes is different. No editing is involved. There is thus no risk of harming a child by putting it through a risky experimental procedure. Whether Genomic Predictions particular technique will actually deliver super-healthy children remains to be seen. The principle seems plausible, though. History may therefore look back on this moment as the true beginning of designer babies. And the tool that has made that possible is called GWAS.

GWAS stands for genome-wide association study. It is the endpoint of a historical process that began in the mid-19th century with Gregor Mendel, a Moravian abbot and amateur botanist. Mendel worked out the first set of rules of heredity. This led to the idea of a gene. And that, when allied with the discovery that the material of heredity is a chemical called DNA, which encodes genetic information in the order of its component units, known as nucleotides, led to the idea of a gene being a particular piece of DNA that carries in its nucleotides the blueprint of a particular protein. This protein goes on to contribute, in combination with environmental effects such as nutrition, to a particular bodily or behavioural characteristic, known as a phenotypic trait.

Since the 1950s, researchers have tried to quantify the relative contributions of genes and the environment to such traits. Mostly, this is in the context of disease. But behavioural characteristics, personality and cognitive ability have also been matters of interest. GWAs expands this process by looking not just at the effects of individual genes, but across the whole genomefor protein-coding genes make up only about 2% of a persons DNA.

Comparisons, over several generations of a family, of the prevalence of a particular trait yield estimates of its heritabilitya measure of how well individual genetic differences account for variations in that trait in a given population. A heritability of 100% indicates that any differences in a trait between individuals in that population are accounted for solely by genetic factors, while 0% suggests the environment alone is responsible. The phrase given population is important. Some populations may be exposed to relevant environmental variables unknown to others. Conversely, genetic factors present in one group (better response to oxygen scarcity in those evolved to live at high altitude, for example) may be absent in another.

An analysis published in 2015 of more than 2,700 studies of heritability shows that its average value, for all traits looked into in those studies, is about 50%. That includes physical traits like susceptibility to heart disease (44%) and eye disorders (71%), and mental ones, including higher-level cognitive functions (47%) such as problem-solving and abstract thought.

Other, less obvious traits are heritable, too. The amount of time a child spends watching television was assumed for many years to have a heritability close to zero. In 1990, however, a study led by Robert Plomin, now at Kings College, London, compared the habits of adopted children with those of their birth mothers. It found television-watching has a heritability of about 45%. Similar surprisingly heritable traits include a childs tendency to be bullied at school (more than 70%) or to be accident-prone (51%). Even someones likelihood of being religious (30-40%) or of getting divorced (13%) is heritable.

In 1989 James Watson, the first head of the Human Genome Project, summarised the mood of many by declaring that We used to think our fate was in our stars. Now we know, in large measure, our fate is in our genes. There was hope then that the genome project would locate those genes. No one was naive enough to think that there existed, say, such a thing as a gene for television-watching. But it was reasonable to believe that there might be a handful of genes which combined to encourage television-watching indirectly. More important, there was an expectation that the heritable causes of things like heart disease might be pinned down to such genetic handfuls. These might then be investigated as drug targets. To everyones frustration, though, few such genes revealed themselves. And in most cases the contributions they made to a conditions heritability were small. Where, then, was the missing heritability?

With hindsight, the answer was obvious. The number of variants that play a role in disease risk is far higher than Mendel-blinded researchers had imagined. Though human beings are genetically more than 99.9% alike, they have 6bn genetic letters in their genomes. This is where the SNPs are hidden, for a diversity of less than 0.1% still leaves room for millions of them. And when SNPs contributions are combined, their effects can be significant. For height, for example, the number of relevant SNPs is reckoned to be about 100,000each adding or subtracting, on average, 0.14mm to or from a persons adult stature. Furthermore, most of these SNPs are in parts of the genome that do not encode proteins at all. Rather, they regulate the activities of other genes and often have no obvious connection to the trait in question.

To be fair, it was mainly human geneticists who were captivated by the simple Mendelian model of single genes with big effects. According to Peter Visscher of the University of Queensland, Australia, many plant and animal scientists knew of traits genetic complexity long before the Human Genome Project started. But they were more interested in breeding better crops or livestock than in understanding the biology behind such complexity.

Dr Visscher was one of the first to realise that human studies would need to recruit more participants and screen for many thousands more SNPs if they were to capture in full the genetic components of most traits. In 2007 he and his colleagues used models to show that for a condition with a prevalence of 10% in the general population, approximately 10,000 volunteers are required to identify the SNPs marking the 5% of those at highest risk of developing that condition. Earlier studies, often with just a few hundred participants, had simply not been powerful enough to see what was going on. And thus was GWAS born.

Ideally, a GWAS would obtain a full sequence of the genome of every participating individual. However, even though the cost of such sequences has fallen dramatically since the completion of the genome project, to about $1,000 a shot, this would still be prohibitively expensive. Instead, researchers use devices called SNP arrays. These detect hundreds of thousands of the most common SNPs for a price of $50 or so.

A combination of SNP arrays, larger samples of volunteers and better computing methods means it is now possible to find millions of variants that contribute to a trait. An individuals score from these variants, known as his polygenic score, can then be calculated by adding up their contributions to give, for example, his risk of developing a particular disease in later life.

Another advance has been a change in the way volunteers are recruited. Institutions called biobanks have come into existence. These hold both tissue samples from, and a range of medical and other data about, large numbers of people who have agreed to make those data available to researchers who meet the criteria employed by the bank in question.

Among the largest of these repositories is the UK Biobank, in Britain. This has 500,000 depositors. One study that drew on it, published in 2018 by Sekar Kathiresan of the Massachusetts General Hospital in Boston and his colleagues, worked out polygenic risk scores for five diseases, including coronary heart disease and type 2 diabetes. By totting up scores from over 6m genetic variants, they were able to elucidate SNP patterns that identify those who are at a threefold higher risk or worse than the general British population of developing one of these diseases. For heart disease, 8% of the population are at such risk. For type 2 diabetes, 3.5%.

Nasim Mavaddat of the University of Cambridge and her colleagues have similarly calculated polygenic risk scores for breast cancer. These showed that a British womans average ten-year risk of developing breast cancer at the age of 47 (the earliest that Englands National Health Service begins screening for the disease) is 2.6%. The study also found that the 19% of women who had the highest risk scores reached this level of risk by the age of 40. Conversely, the 10% at lowest risk did not cross the threshold until they were 80.

Using these and similar studies, it is possible to draw up lifetime risk profiles for various medical conditions. A British firm called Genomics has done that for 16 diseases (see chart). This will help screening programmes to triage who they screen, by offering their services earlier to those at high risk of developing a condition early in their lives. It will also permit the dispensing of risk-appropriate advice about diet and exercise to those who need it most, and the early offering to those who might benefit from them of things like statins and antihypertensive drugs. In light of all this Englands National Health Service announced in July that 5m healthy Britons would be offered free gene tests.

A third study that drew on the UK Biobank is rather different. It was published in October and demonstrated the power of GWAS to reach beyond non-medical matters. It examined patterns of internal migration in Britain, and showed that there has been an outward migration from former coalmining areas of people with SNP patterns associated with high educational attainmentprecisely the sorts of individuals economically deprived places can least afford to lose.

Educational attainment also demonstrates how heritability varies with environment. In Norway, for example, heritability of educational attainment increased after the second world war as access to education widened. Since all children now had more or less the same opportunities at school, environmental variation was largely ironed out and the effects of genetic differences consequently exaggerated.

Both of these examples foreshadow how the sort of genetics made possible by GWAS can have political consequences. The implication of the internal-migration study is that the geographically left-behind are dimmer, on average, than the leavers. The implication of the Norwegian study might likewise be seen by some as suggesting that those who have done well at school and thus snagged the best (and best-paid) jobs are part of a genetic elite that deserves its success, rather than being the lucky winners of a genetic lottery.

And that is just within a country. Start comparing people from different parts of the world and you enter a real minefield. Because most of the genetic data now available come from populations of European ancestry, their predictive power is poorer for people from elsewhere. Alicia Martin of the Broad Institute in Massachusetts and her colleagues scored West Africans for height based on SNPs drawn from studies on European or European-derived populations. The scores predicted that West Africans should be shorter than Europeans. Actually, they are not.

As more people of non-European ancestry are sequenced, these problems may abate. But if group-based differences emerge or persist in the face of better data, that would be cause for concern. Differences between groups in things like height are rarely cause for prejudice beyond a jocular level. For something like educational attainment, by contrast, there is a risk that politically motivated groups would try to exploit any differences found to support dubious theories of racial superiority.

To some historians, this looks horribly familiar. They fear that the old spectre of eugenics risks rising in a new guise. As Nathaniel Comfort of Johns Hopkins University, in Baltimore, observes, The IQ test was invented in order to identify students who needed extra help in school. But within about a decade, it was being used as a tool to weed out the so-called feebleminded, not just from school but from the gene pool. Such fears of genetic stratification would become particularly acute if polygenic scores were applied to embryos for the purpose of selecting which to implant during IVFas Genomic Prediction is just about to do.

Genomic Prediction and a second firm, MyOme (which is not yet accepting customers), claim to be able to build up an accurate picture of an embryos genome. That is tricky because the sequencing has to be carried out using the tiny quantities of DNA in a few cells taken from that embryo. A sequence so obtained would normally be full of errors. The two companies say they can deal with this by comparing embryonic sequences with those of the biological parents. All of the DNA in the embryo has come from one or other parent, so blocks of embryonic DNA can be matched to well-established sequences from their parental progenitors and an accurate embryonic sequence established. That makes working out the embryos SNP pattern possible.

Genomic Prediction thus says it is able to offer couples undergoing IVF a polygenic risk score for each embryo for a variety of diseases including type 1 diabetes, type 2 diabetes, breast cancer, testicular cancer, prostate cancer, basal-cell carcinoma, malignant melanoma, heart attack, atrial fibrillation, coronary artery disease, hypertension and high cholesterol. At the moment it does not offer scores for non-medical traits like height or educational attainment. But there is nothing to prevent it from doing so should it so wish.

Even for medically relevant scores, however, some worry about this approach. One concern is pleiotropythe phenomenon of the same piece of DNA influencing several apparently unrelated traits. Choosing an embryo with a low risk of heart disease might accidentally give it, say, a higher chance of developing epilepsy. Single-mindedly maximising scores for positive traits like intelligence or height may therefore increase the risk of genetic disorders.

Stephen Hsu of Michigan State University, one of Genomic Predictions founders, acknowledges the theoretical risk of this, but argues that serious pleiotropic effects are unlikely. If you looked at a bunch of kids with IQs of, say, 160 or 170, he says, I doubt youd find much seriously wrong with them. Theyd just be a bunch of geeks. Dr Hsu, who in 2014 predicted that reproductive technologies would soon be used to select for more intelligent offspring, estimates that an IQ gain of between 10 and 15 points would be possible if couples were allowed to choose between ten embryos. He also thinks that further gains would probably accumulate if people selected in this way went on to select their own offspring on the basis of intelligence.

This is plausible. Before 2008, when the first SNP chips for cattle became available, the annual milk yield of dairy cows in America had been increasing at about 50kg per year. After six years of chip-based polygenic selection, the rate of increase had doubled to more than 100kg per year. This suggests the technique is powerfulin cattle at least. Despite Dr Hsus optimism, however, pleiotropism has reared its head in these animals. They have become less fertile and have weaker immune systems.

In the end, then, it is generally a good idea to remember that human beings have already been optimised by a powerful agent called natural selection. Trade-offs between different pieces of physiology, even in domestic animals, will have been forged in the crucible of evolution and will generally be optimal, or close to it. Genetic tinkering may sometimes improve things. But by no means always.

Read more:
Modern genetics will improve health and usher in designer children - The Economist

Recommendation and review posted by Bethany Smith

Although hair loss may seem like a more prominent problem in men, women are nearly as likely to lose or have thinning hair. Most women notice it in their 50s or 60s, but it can happen at any age and for a variety of reasons.

Hair grows in three different cycles: anagen, catagen, and telogen. About 90% of the hair on the head is in the anagen, or growth phase, which lasts anywhere from 2 to 8 years. The catagen, or transition phase, typically lasts 2-3 weeks, during which the hair follicle shrinks. During the telogen cycle, which lasts around 2 to 4 months, the hair rests. An overwhelming majority of the time, the hair is on the scalp; it is growing. Only about 10% of the strands are in transition or resting at any one time. Hair grows about 6 inches a year for most people.

The most common cause of hair loss is a hereditary condition called male-pattern hair loss or female-pattern hair loss. It usually occurs gradually with aging and in predictable patterns a receding hairline and bald spots in men and thinning hair in women. For women, hair loss is most common around ages 45 to 55 but can commence as early as a woman in her 20s. Over half of women will find their hair is thinning by the age of 50. For men, it can happen as early as their teens, and it starts with the hairline receding. Women may notice their part widening or the volume decreasing in the middle of their scalp. When it comes to seeking treatment, women will usually take action far more quickly than men.

Most people lose anywhere from 50 to 100 strands of hair each day, according to the American Academy of Dermatology. On the days when hair is washed, people can lose up to 250 strands. But dont avoid washing in an attempt to keep the hair, as it will fall out eventually, anyway.

For those who dont plan on counting their hair every day, there are ways to know when hair is thinning or being lost at a higher rate. When waking up in the morning, there may be an usually large amount on your pillow. Or, when you comb your hair (especially without tugging, which can pull the hair out), more than normal will be left in the comb. Another way to diagnose whether a woman is experiencing excessive hair loss is through family history. What did your mother, aunts, or grandmothers look like at similar ages? If they had similar amounts of hair loss at such ages, it could be a telltale sign of female pattern hair loss. Typically, each time a normal hair follicle is shed, it is replaced by hair that is equal in size. But in women with female pattern hair loss, the new hair is finer and thinner -a more miniaturized version of itself. The hair follicles are shrinking, and eventually, they quit growing altogether.

If hair loss is sudden or excessive, it is likely to be caused by something other than heredity, like a medical condition. There is a wide range of conditions that can bring on hair loss, with some of the most common being pregnancy, thyroid disorders, and anemia. Others include autoimmune diseases, polycystic ovary syndrome (PCOS), and skin conditions such as psoriasis and seborrheic dermatitis. There also may be a link between menopause and hair loss.

Menopause is also a pretty common culprit in female hair loss and is one of the many symptoms that come about due to the major hormonal fluctuations that occur at this time in a womans life. Menopausal hair loss usually manifests as a general thinning all over the scalp, instead of hugely noticeable bald spots.

Other reasons for hair loss include extreme stress, physical trauma like surgery or intense illness, dramatic weight loss over a short period of time; and taking too much or too little of certain vitamins. And hair loss can occur a couple of weeks to six months after any of these experiences.

Regular, garden variety female pattern baldness is age-related and the most common cause of female hair loss and can occur even in non-menopausal women. This is a genetic condition that is also known as androgenetic alopecia. It is quite normal not that that is of much comfort and is usually confined to overall thinning rather than patchy losses.

One other way to thin hair is self-inflicted -hairstyles like cornrows or too-tight braids can cause hair loss called traction alopecia. All of the things women do to manipulate their hair dyes, chemical treatments, bad brushes, blow dryers, and flat irons can result in damage and breakage. This includes brushing too much and towel drying aggressively when the hair is wet. Luckily, for most of these issues, the hair grows back, or the loss can be reversed with medical treatments. But it is important to see a dermatologist if there seems to be something wrong, because the sooner treatment is started, the better the chances are for improving your growing season. Another thing to keep in mind is that wearing your hair in a tight hairstyle such as a heavy ponytail or bun or tight braids can cause hair loss too.

Well, it mostly depends on the reason for it in the first place. A dietary deficiency can often be corrected with supplements or an improvement in what you eat. Life-stage issues such as pregnancy and menopause are going to take their toll, and quite often, the level of hair loss will even out once the stage is complete. In the case of menopause, that full, lustrous head of hair probably wont return to what it once was, but the hair fall will usually slow dramatically. Hormone replacement therapy can help, although if hair loss is the only symptom worrying you and not too traumatically you may want to forgo the HRT and muddle through.

Just as everybody responds differently to their hair loss, everybody responds differently to treatments for it. Its critical to find out the cause first, so that could mean a visit to your doctor. Once you know what youre dealing with, start there. Maybe you need treatment for stress, for a hormonal imbalance, or for PCOS or another medical condition. But if you find out you have female pattern hair loss, there are certainly treatments that can help manage it. What are some of the most effective treatments/strategies for women with hair loss? Again, the solution for you will depend on your situation, but here are some strategies you might want to look into. It depends on the cause and on the individual, but some of the strategies include:

Minoxidil. Initially developed as a treatment for high blood pressure, it became known as a drug that could grow hair in places where it was lost. Some women not all experience great results. Its important to keep in mind that one must stay on Minoxidil for at least six months to cover the full hair growth cycle. One favorite is Hair Restoration Laboratories Ultra Strength Hair Regrowth Treatment which not only contains FDA-approved 5% minoxidil but also a number of powerful DHT-blocking ingredients that, when combined with 5% minoxidil, significantly improve the quality of hair and scalp.

Platelet Rich Plasma Therapy (PRP). Known as the Vampire treatment, the patients blood is drawn, naturally enhanced to boost the number of platelets and key growth factors, then reinjected back into the scalp. This can revive dying follicles and hair.

Iron supplements Iron deficiency is thought to be a hair loss cause in a lot of women. If women are experiencing hair loss, shedding or thinning, it is highly recommended that they see their doctor for a blood test to check levels, particularly if theyre vegan/vegetarian, have a history of anemia or experience heavy menstrual bleeding. The doctor may prescribe an iron supplement, which could help to remediate the hair loss.

Hair Restoration Laboratories Hair Restore Shampoo and Conditioner Set. This shampoo and conditioner set helps keep hair healthy and protects against the effects that cause hair loss. Combined, the Hair Restore Shampoo and Conditioner Set contains over 30 potent ingredients formulated to fight DHT (the primary factor the causes female pattern hair loss), prevent hair loss and thinning, and stimulate thicker, fuller and healthier hair regrowth.

If you are suffering from hair loss or thinning, the most likely culprit is genetics. While you cannot fight your genetics, there are numerous treatments available to effectively prevent any further loss or thinning and restore the hair that you have already lost. Time is not on your side with hair loss. So, the sooner you consult with a physician to identify the causes, the sooner you will be able to give your hair what it needs to grow strong, thick, and healthy.

Excerpt from:
Female Hair Loss And What To Do About It - Explosion

Recommendation and review posted by Bethany Smith

Projected Lifetime Healthcare Costs of High-Risk Patients on Conventional Medical Therapy, such as Statins, Were Compared with and without the Cardiovascular Risk Reduction Demonstrated with Icosapent Ethyl in the REDUCE IT Study

Analysis Accounted Patient Treatment Outcomes as Well as U.S. Private Insurance and Medicare Costs

DUBLIN, Ireland and BRIDGEWATER, N.J., Nov. 11, 2019 (GLOBE NEWSWIRE) -- Amarin Corporation plc (NASDAQ: AMRN) announced today a summary of results from a patient-level, cost-effectiveness analysis of icosapent ethyl (Vascepa).1 This comprehensive analysis evaluated the cost-effectiveness of icosapent ethyl in reducing cardiovascular (CV) risk among high-risk patients as demonstrated in the landmark REDUCE-IT2cardiovascular outcomes study. In this newly reported analysis, use of icosapent ethyl was projected to not only be cost-effective but also to reduce long-term health care costs in a majority of the scenarios analyzed.

The findings were disclosed in an abstract titled, Cost-Effectiveness of Icosapent Ethyl in REDUCE-IT, in connection with the 2019 Scientific Sessions of the American Heart Association (AHA), scheduled for November 16 18 in Philadelphia, PA. William S. Weintraub, M.D., director of Outcomes Research with MedStar Cardiovascular Research Network, and lead author of the analysis, is scheduled to present the results in more detail at AHA on Saturday, November 16, at 7:30 a.m.

Dr. Weintraub and the MedStar Cardiovascular Research Network, known for conducting thoughtful pharmacoeconomic analysis, used available data for cost information for treatment and rehabilitation of patients from stroke, myocardial infarction, revascularization, hospitalization and cardiovascular death and assessed how such costs decline relative to the cost of patient treatment with icosapent ethyl based on the results of the REDUCE-IT study.

Background: Despite statin therapy and well-controlled LDL-C, many high CV risk patients continue to experience CV events. This persistent CV risk was evaluated via REDUCE-IT, which enrolled statin-treated patients with controlled LDL-C (>40 - 100 mg/dL) and elevated triglycerides (135 - <500 mg/dL), with established CV disease or diabetes combined with other CV risk factors. Over 4.9 years median follow-up, REDUCE-IT showed that icosapent ethyl lowered risk of first and total CV events by 25% and 30%, respectively.

In REDUCE-IT, adverse events occurring with icosapent ethyl use at greater than 5% and greater than placebo were: peripheral edema (6.5% Vascepa versus 5.0%), although there was no increase in the rate of heart failure in Vascepa patients; constipation (5.4% Vascepa versus 3.6%), although mineral oil, as used as placebo, is known to lower constipation; and atrial fibrillation (5.3% Vascepa versus 3.9%), although there were reductions in rates of cardiac arrest, sudden death and myocardial infarctions observed in Vascepa patients. More information on safety data associated with REDUCE-IT is provided further below.

Methods: The analysis applied treatment effects from REDUCE-IT, health care costs from national sources, including private insurance and Medicare, and conducted a combination cost-effectiveness analysis utilizing both patient-level in-trial cost and clinical outcomes and long-term costs, events and life expectancy derived from Markov simulation models. The model projected lifetime healthcare costs, CV events, survival and quality-adjusted life-years (QALYs) for icosapent ethyl versus placebo in REDUCE-IT eligible patients.

Results: Icosapent ethyl was a dominant strategy (i.e., cost saving) in 70% of simulations, offering the rare finding of better outcomes at lower healthcare costs. In probabilistic sensitivity analysis, >85% of simulations indicated that icosapent ethyl would be cost-effective (i.e., below $50,000 per QALY gained) compared with placebo.

Conclusion: In this combined patient-level and simulation cost-effectiveness analysis, icosapent ethyl in high CV risk patients shows exceptional benefit with CV event reduction as well as cost-savings in-trial and over patients lifetime in the majority of simulations.

John Thero, president and chief executive officer of Amarin, developer of Vascepa, commented, This analysis helps to validate something weve long believed, and that is central to our mission. It is possible to deliver significant innovation that meaningfully addresses our nations most prevalent and costly health epidemic, reduces impacts on patients and families, and drives down costs longer term in the health system. We are working to make this therapy broadly available for improved patient care in high risk patients subject to appropriate regulatory review.

Other Considerations:MedStar Cardiovascular Research Network (MedStar), which conducted this cost-effectiveness analysis, led by its director of Outcomes Research, Dr. Weintraub, is an organization dedicated to fighting heart disease, stroke and other conditions affecting the heart and blood vessels and is affiliated with MedStar Health Research Institute and Medstar Health. Amarin funded this analysis.

The patient cost and actuarial information used by MedStar in this cost-effective analysis were derived from sources independent of Amarin. Data regarding cardiovascular risk reduction demonstrated by Vascepa was sourced from previously published information from the total cohort of the REDUCE-IT study (not from the REDUCE-IT USA cohort published today in Circulation3 which showed even more pronounced results albeit subject to the limitations of subset analysis) with support provided by Amarin in making the data available to MedStar for analysis. Included in this data was outcomes results pertaining to first occurrences of cardiovascular events and recurrent events. As disclosed in the publication of the recurrent events analysis in the Journal of the American College of Cardiology4, the analyses addressed tertiary or exploratory endpoints using a series of statistical models, most of which were prespecified and one of which was post hoc. Each recurrent event statistical model has inherent strengths and weaknesses, with no single model considered definitive or outperforming the other models, as this is an evolving field of science. Nonetheless, results from the total primary and total key secondary endpoint events analyses are consistent across the various recurrent event statistical models and are also consistent with the original primary and secondary endpoint results.

Furthermore, the cost-effectiveness analysis conducted by MedStar, as is typical of cost-effectiveness analyses, was not prespecified. Amarin funded MedStar because of its expertise to conduct this analysis, as opposed to Amarin conducting it on its own, to mitigate perceptions of potential bias inherent in post hoc analyses.

This press release is timed today pursuant to AHAs release of an abstract for this presentation. It is Amarins understanding that AHA plans to release abstracts for some but not all (e.g. not any presentation deemed a late breaker, such as the presentation regarding EVAPORATE) of the seven scientific presentations Amarin listed in its press release dated November 4, 2019. Without potentially jeopardizing presentation at AHA, Amarin looks forward to communications regarding all of these presentations pursuant to their presentation at AHA.

About AmarinAmarin Corporation plc. is a rapidly growing, innovative pharmaceutical company focused on developing therapeutics to improve cardiovascular health. Amarins product development program leverages its extensive experience in polyunsaturated fatty acids and lipid science. Vascepa (icosapent ethyl) is Amarin's first FDA-approved drug and is available by prescription in the United States, Lebanon and the United Arab Emirates. Amarins commercial partners are pursuing additional regulatory approvals for Vascepa in Canada, China and the Middle East. For more information about Amarin, visit http://www.amarincorp.com.

About REDUCE-IT REDUCE-IT, an 8,179-patient cardiovascular outcomes study, was completed in 2018. REDUCE-IT was the first multinational cardiovascular outcomes study that evaluated the effect of prescription pure EPA therapy as an add-on to statins in patients with high cardiovascular risk who, despite stable statin therapy, had elevated triglyceride levels (at least 135 mg/dL). A large proportion of the male and female patients enrolled in this outcomes study were diagnosed with type 2 diabetes.

More information on the REDUCE-IT study results can be found at http://www.amarincorp.com.

About Cardiovascular DiseaseWorldwide, cardiovascular disease (CVD) remains the #1 killer of men and women. In the United States CVD leads to one in every three deaths one death approximately every 38 seconds with annual treatment cost in excess of $500 billion.5,6

Multipleprimary and secondary preventiontrials have shown a significant reduction of 25% to 35% in the risk ofcardiovascular eventswithstatintherapy, leaving significant persistent residual risk despite the achievement of target LDL-C levels.7

Beyond the cardiovascular risk associated with LDL-C, genetic, epidemiologic, clinical and real-world data suggest that patients with elevated triglycerides (TG) (fats in the blood), and TG-rich lipoproteins, are at increased risk for cardiovascular disease.8,9,10,11

About Vascepa (icosapent ethyl) CapsulesVascepa (icosapent ethyl) capsules are a single-molecule prescription product consisting of the omega-3 acid commonly known as EPA in ethyl-ester form. Vascepa is not fish oil, but is derived from fish through a stringent and complex FDA-regulated manufacturing process designed to effectively eliminate impurities and isolate and protect the single molecule active ingredient from degradation. Vascepa, known in scientific literature as AMR101, has been designated a new chemical entity by the FDA. Amarin has been issued multiple patents internationally based on the unique clinical profile of Vascepa, including the drugs ability to lower triglyceride levels in relevant patient populations without raising LDL-cholesterol levels.

The FDA has not completed its review and made a final determination on a supplemental new drug application related to REDUCE IT. FDA has not reviewed the information herein or determined whether to approve Vascepa for use to reduce the risk of major adverse cardiovascular events in the REDUCE-IT patient population.

Indication and Usage Based on Current FDA-Approved Label (not including REDUCE-IT results)

Important Safety Information for Vascepa Based on Current FDA-Approved Label (not including REDUCE-IT results) (Includes Data from Two 12-Week Studies (n=622) (MARINE and ANCHOR) of Patients with Triglycerides Values of 200 to 2000 mg/dL)

FULL VASCEPA PRESCRIBING INFORMATION CAN BE FOUND AT http://WWW.VASCEPA.COM.

Important Safety Information for Vascepa based on REDUCE-IT, as previously reported in The New England Journal of Medicine publication of the primary results of the REDUCE-IT study:

Important Cautionary Information About These DataFurther REDUCE-IT data assessment and data release are expected to yield additional useful information to inform greater understanding of the trial outcome. For example, detailed data assessment by regulatory authorities, such as the FDA and Health Canada, will continue and take time to complete and announce. The FDA advisory committee process and the final evaluation by regulatory authorities of the totality of efficacy and safety data from REDUCE-IT is anticipated to include some or all of the following, as well as other considerations: new information or analyses affecting the degree of treatment benefit on studied endpoints; study conduct and data robustness, quality, integrity and consistency; additional safety data considerations and risk/benefit considerations; and consideration of REDUCE-IT results in the context of other clinical studies. More detailed presentation of such considerations is set forth in the risk factors section of Amarins Quarterly Report on Form 10-Q filed with the U.S. Securities and Exchange Commission. Because regulatory reviews are typically fluid and not definitive interactions between sponsor and agency on individual elements of an application and related information, Amarin does not plan to update investors further on ongoing communications with regulatory authorities. Amarin plans to announce the final outcome of such regulatory reviews when appropriate.

Forward-Looking StatementsThis press release contains forward-looking statements, including statements regarding the use of Vascepa to potentially help millions of patients and projections related to the cost-effectiveness of Vascepa. These forward-looking statements are not promises or guarantees and involve substantial risks and uncertainties. Among the factors that could cause actual results to differ materially from those described or projected herein include the following: uncertainties associated generally with research and development, clinical trials and related regulatory reviews and approvals; the risk that data interpretations or other information from third parties, the regulatory review process, regulatory authorities and in connection with an advisory committee could be made public that are negative or may delay approval or limit Vascepas marketability; the risk that special protocol assessment (SPA) agreements with the FDA are not a guarantee that FDA will approve a product candidate; the risk associated with the FDA's rescinding the REDUCE-IT SPA agreement; the risk related to FDA advisory committee meetings; and the risk that the FDA may not complete its review of the REDUCE-IT sNDA within the timing expected. A further list and description of these risks, uncertainties and other risks associated with an investment in Amarin can be found in Amarin's filings with the U.S. Securities and Exchange Commission, including its most recent Quarterly Report on Form 10-Q. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Amarin undertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise.

Availability of Other Information About AmarinInvestors and others should note that Amarin communicates with its investors and the public using the company website (www.amarincorp.com), the investor relations website (investor.amarincorp.com), including but not limited to investor presentations and investor FAQs, Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Amarin posts on these channels and websites could be deemed to be material information. As a result, Amarin encourages investors, the media, and others interested in Amarin to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Amarins investor relations website and may include social media channels. The contents of Amarins website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.Amarin Contact Information

Investor Inquiries:Elisabeth SchwartzInvestor RelationsAmarin Corporation plcIn U.S.: +1 (908) 719-1315 investor.relations@amarincorp.com

Lee M. SternSolebury TroutIn U.S.: +1 (646) 378-2992 lstern@soleburytrout.com

Media Inquiries:Gwen FisherCorporate Communications Amarin Corporation plcIn U.S.: +1 (908) 325-0735 pr@amarincorp.com

References_______________1 William S Weintraub, MedStar Washington Hosp Ctr, Washington, DC; Deepak L Bhatt, Brigham and Women's Hosp, Boston, MA; Zugui Zhang, Christiana Care Health System, Newark, DE et al., Cost-Effectiveness of Icosapent Ethyl in REDUCE-IT.2 Bhatt DL, Steg PG, Miller M, et al. Cardiovascular Risk Reduction with Icosapent Ethyl for Hypertriglyceridemia. N Engl J Med 2019;380:11-223 Bhatt DL, Miller M, Brinton EA, et al. REDUCE-IT USA: Results from the 3,146 Patients Randomized in the United States. Circulation 2019. DOI: 10.1161/CIRCULATIONAHA.119.044440.4 Bhatt DL, Steg PG, Miller M, et al. Effects of Icosapent Ethyl on Total Ischemic Events: From REDUCE-IT. J Am Coll Cardiol 2019; 73:2791-28025 American Heart Association. 2018. Disease and Stroke Statistics-2018 Update.6 American Heart Association. 2017. Cardiovascular Disease: A Costly Burden for America Projections Through 2035.7 Ganda OP, Bhatt DL, Mason RP, et al. Unmet need for adjunctive dyslipidemia therapy in hypertriglyceridemia management. J Am Coll Cardiol. 2018;72(3):330-343.8 Budoff M. Triglycerides and triglyceride-rich lipoproteins in the causal pathway of cardiovascular disease. Am J Cardiol. 2016;118:138-145.9 Toth PP, Granowitz C, Hull M, et al. High triglycerides are associated with increased cardiovascular events, medical costs, and resource use: A real-world administrative claims analysis of statin-treated patients with high residual cardiovascular risk. J Am Heart Assoc. 2018;7(15):e008740.10 Nordestgaard BG. Triglyceride-rich lipoproteins and atherosclerotic cardiovascular disease - New insights from epidemiology, genetics, and biology. Circ Res. 2016;118:547-563.11 Nordestgaard BG, Varbo A. Triglycerides and cardiovascular disease. Lancet. 2014;384:626635.

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New Analysis Shows Icosapent Ethyl (Vascepa) Is Cost Effective and Offers Rare Finding of Better Outcomes at Lower Healthcare Costs When Used to Treat...

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Prespecified Analysis of 3,146 Patients in the USA Enrolled in REDUCE-IT Showed 31% Relative Risk Reductions for First Occurrence of Both 5-Point MACE and 3-Point MACE

Significant Reductions Shown in All Predefined Composite and Individual Cardiovascular Endpoints, Including Cardiovascular Death and All-Cause Mortality

Tolerability and Safety Findings Consistent with Full Study

Results Published Today in Circulation and Scheduled for Presentation at American Heart Association 2019 Scientific Sessions on November 17

DUBLIN, Ireland and BRIDGEWATER, N.J., Nov. 11, 2019 (GLOBE NEWSWIRE) -- Amarin Corporation plc (NASDAQ: AMRN) announced today the results from the subgroup of 3,146 patients randomized in the United States within the global Vascepa (icosapent ethyl) cardiovascular (CV) outcomes trial, REDUCE-IT. This prespecified REDUCE-IT subgroup analysis showed robust risk reductions in the USA patients treated with icosapent ethyl 4 g/day versus placebo across all prespecified composite and individual primary and secondary endpoints, including 31% relative risk reduction and 6.5% absolute risk reduction in first occurrence of 5-point major adverse cardiovascular events (MACE), corresponding to a number needed to treat of 15 (NNT=15), and a significant 30% relative and 2.6% absolute risk reduction (NNT=38) in all-cause mortality in the USA subgroup.

Additional prespecified cardiovascular endpoints in which the REDUCE-IT USA subgroup showed significant relative risk reduction included myocardial infarction, cardiovascular death, and stroke, similar to the full cohort in the overall REDUCE-IT global results.1 These results were incremental to the cardiovascular risk reduction achieved by conventional therapy administered to the high-risk patients studied, including incremental to statin therapy.

In the REDUCE-IT USA subgroup, 3,146 patients (38.5% of the full trial cohort) were randomized and followed for a median of 4.9 years; 32.3% were women, 9.7% Hispanic. USA placebo patients had a higher primary endpoint event rate compared with the full cohort (67.4 versus 57.4 per 1,000 patient-years, respectively). The primary endpoint (5-point MACE) occurred in 24.7% of placebo versus 18.2% of icosapent ethyl patients (HR 0.69, 95% CI 0.59-0.80, p=0.000001); the key secondary endpoint (3-point MACE) occurred in 16.6% of placebo versus 12.1% of icosapent ethyl patients (HR 0.69, 95% CI 0.57-0.83, p=0.00008). All prespecified hierarchical primary and secondary endpoints were significantly reduced in the USA subgroup, including myocardial infarction (8.8% to 6.7%, HR 0.72, 95% CI 0.56-0.93, p=0.01), cardiovascular death (6.7% to 4.7%, HR 0.66, 95% CI 0.49-0.90, p=0.007), stroke (4.1% to 2.6%, HR 0.63, 95% CI 0.43-0.93, p=0.02), and all-cause mortality (9.8% to 7.2%, HR 0.70, 95% CI 0.55-0.90, p=0.004). In the full study cohort, there was a trend towards a reduction in all-cause mortality, with each of these other primary and secondary endpoints also achieving statistical significance in the full study cohort. Safety and tolerability findings in the USA subgroup were consistent with the full study cohort.

REDUCE-IT was not specifically powered to examine individual subgroups. P-values presented for the USA subgroup are nominal and exploratory with no adjustment for multiple comparisons. Differences in efficacy outcomes for the USA patients are best viewed as qualitative and not quantitative; nevertheless, the data are useful and provide reassurance that the results in the USA are at least as strong as the results seen outside the USA and in the trial overall.

The REDUCE-IT USA results are scheduled to be presented on Sunday, November 17 at the 2019 Scientific Sessions of the American Heart Association (AHA) in Philadelphia, PA. The REDUCE-IT USA study results were published today in Circulation, AHAs official scientific journal.2 The global results of REDUCE-IT from the full cohort of the study were previously published in The New England Journal of Medicine for the first occurrence of the studys primary and secondary endpoints and results of the studys full cohort with respect to total events were previously published in The Journal of American College of Cardiology.1,3This newly published data in Circulation is the first publication of detailed results from the REDUCE-IT USA cohort.

Scientific presentation: The presentation of the REDUCE-IT USA results at AHA will be delivered by the Global Principal Investigator and Steering Committee Chair of the study, Deepak L. Bhatt, M.D., M.P.H., executive director of Interventional Cardiovascular Programs at Brigham and Womens Hospital Heart and Vascular Center, and professor of medicine at Harvard Medical School. Dr. Bhatts featured presentation, titled REDUCE-IT USA: Results from the 3,146 Patients Randomized in the United States, will be delivered on November 17, 4:20 - 4:25 p.m.

Dr. Bhatt stated: The REDUCE-IT USA results confirm the findings of the global REDUCE-IT trial and further highlight the importance of the prevention of residual, or persistent, risk of cardiovascular events in statin-treated patients with only moderately increased triglycerides. The USA subgroup, which had more risk factors than the overall REDUCE-IT population, experienced particularly robust risk reduction from the use of icosapent ethyl in preventive cardiovascular care, including a statistically significant 30% reduction in death. These findings are also remarkable when you consider that in some multinational cardiovascular trials, patients in the United States experience less benefit.

Amarin perspective The results of the REDUCE-IT USA subgroup are further evidence of the robust and consistent nature of this landmark study and its applicability to typical clinical practice in the United States, stated Steven Ketchum, Ph.D., president of research and development and chief scientific officer of Amarin. We believe that these very impressive results further validate that persistent cardiovascular risk beyond cholesterol management can be significantly reduced with Vascepa in the high-risk patient population studied in REDUCE-IT.

The REDUCE-IT USA subgroup analysis was funded by Amarin. Dr. Bhatt receives research funding from Amarin that goes to Brigham and Womens Hospital.

About AmarinAmarin Corporation plc. is a rapidly growing, innovative pharmaceutical company focused on developing therapeutics to improve cardiovascular health. Amarins product development program leverages its extensive experience in polyunsaturated fatty acids and lipid science. Vascepa (icosapent ethyl) is Amarin's first FDA-approved drug and is available by prescription in the United States, Lebanon and the United Arab Emirates. Amarins commercial partners are pursuing additional regulatory approvals for Vascepa in Canada, China and the Middle East. For more information about Amarin, visit http://www.amarincorp.com.

About REDUCE-IT REDUCE-IT, an 8,179-patient cardiovascular outcomes study, was completed in 2018. REDUCE-IT was the first multinational cardiovascular outcomes study that evaluated the effect of prescription icosapent ethyl (IPE) as an add-on to statins in patients with high cardiovascular risk who, despite stable statin therapy, had elevated triglyceride levels (at least 135 mg/dL). A large proportion of the male and female patients enrolled in this outcomes study were diagnosed, prior to study enrollment, with type 2 diabetes.

More information on the REDUCE-IT study results can be found at http://www.amarincorp.com.

About Cardiovascular DiseaseWorldwide, cardiovascular disease (CVD) remains the #1 killer of men and women. In the United States CVD leads to one in every three deaths one death approximately every 38 seconds with annual treatment costs in excess of $500 billion.4,5

Multipleprimary and secondary preventiontrials have shown a significant reduction in the risk ofcardiovascular eventswithstatintherapy, leaving significant persistent residual risk despite the achievement of target LDL-C levels.6

Beyond the cardiovascular risk associated with LDL-C, genetic, epidemiologic, clinical and real-world data suggest that patients with elevated triglycerides (TG) (fats in the blood), and TG-rich lipoproteins, are at increased risk for cardiovascular disease.7,8,9,10

About Vascepa (icosapent ethyl) CapsulesVascepa (icosapent ethyl) capsules are a single-molecule prescription product consisting of the omega-3 acid commonly known as EPA in ethyl-ester form. Vascepa is not fish oil, but is derived from fish through a stringent and complex FDA-regulated manufacturing process designed to effectively eliminate impurities and isolate and protect the single molecule active ingredient from degradation. Vascepa, known in scientific literature as AMR101, has been designated a new chemical entity by the FDA. Amarin has been issued multiple patents internationally based on the unique clinical profile of Vascepa, including the drugs ability to lower triglyceride levels in relevant patient populations without raising LDL-cholesterol levels.

The FDA has not completed its review and made a final determination on a supplemental new drug application related to REDUCE-IT. FDA has not reviewed the information herein or determined whether to approve Vascepa for use to reduce the risk of major adverse cardiovascular events in the REDUCE-IT patient population.

Indication and Usage Based on Current FDA-Approved Label (not including REDUCE-IT results)

Important Safety Information for Vascepa Based on Current FDA-Approved Label (not including REDUCE-IT results) (Includes Data from Two 12-Week Studies (n=622) (MARINE and ANCHOR) of Patients with Triglycerides Values of 200 to 2000 mg/dL)

FULL VASCEPA PRESCRIBING INFORMATION CAN BE FOUND AT http://WWW.VASCEPA.COM.

Important Safety Information for Vascepa based on REDUCE-IT, as previously reported in The New England Journal of Medicine publication of the primary results of the REDUCE-IT study:

Important Cautionary Information About These DataFurther REDUCE-IT data assessment and data release are expected to yield additional useful information to inform greater understanding of the trial outcome. For example, detailed data assessment by regulatory authorities, such as the FDA and Health Canada, will continue and take time to complete and announce. The FDA advisory committee process and the final evaluation by regulatory authorities of the totality of efficacy and safety data from REDUCE-IT is anticipated to include some or all of the following, as well as other considerations: new information or analyses affecting the degree of treatment benefit on studied endpoints; study conduct and data robustness, quality, integrity and consistency; additional safety data considerations and risk/benefit considerations; and consideration of REDUCE-IT results in the context of other clinical studies. More detailed presentation of such considerations is set forth in the risk factors section of Amarins Quarterly Report on Form 10-Q filed with the U.S. Securities and Exchange Commission. Because regulatory reviews are typically fluid and not definitive interactions between sponsor and agency on individual elements of an application and related information, Amarin does not plan to update investors further on ongoing communications with regulatory authorities. Amarin plans to announce the final outcome of such regulatory reviews when appropriate.

Forward-Looking StatementsThis press release contains forward-looking statements, including statements regarding the use of Vascepa to potentially help millions of patients. These forward-looking statements are not promises or guarantees and involve substantial risks and uncertainties. Among the factors that could cause actual results to differ materially from those described or projected herein include the following: uncertainties associated generally with research and development, clinical trials and related regulatory reviews and approvals; the risk that data interpretations or other information from third parties, the regulatory review process, regulatory authorities and in connection with an advisory committee could be made public that are negative or may delay approval or limit Vascepas marketability; the risk that special protocol assessment (SPA) agreements with the FDA are not a guarantee that FDA will approve a product candidate; the risk associated with the FDA's rescinding the REDUCE-IT SPA agreement; the risk related to FDA advisory committee meetings; and the risk that the FDA may not complete its review of the REDUCE-IT sNDA within the timing expected. A further list and description of these risks, uncertainties and other risks associated with an investment in Amarin can be found in Amarin's filings with the U.S. Securities and Exchange Commission, including its most recent Quarterly Report on Form 10-Q. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Amarin undertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise.

Availability of Other Information About AmarinInvestors and others should note that Amarin communicates with its investors and the public using the company website (www.amarincorp.com), the investor relations website (investor.amarincorp.com), including but not limited to investor presentations and investor FAQs, Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Amarin posts on these channels and websites could be deemed to be material information. As a result, Amarin encourages investors, the media, and others interested in Amarin to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Amarins investor relations website and may include social media channels. The contents of Amarins website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.Amarin Contact Information

Investor Inquiries:Elisabeth SchwartzInvestor RelationsAmarin Corporation plcIn U.S.: +1 (908) 719-1315 investor.relations@amarincorp.com

Lee M. SternSolebury TroutIn U.S.: +1 (646) 378-2992 lstern@soleburytrout.com

Media Inquiries:Gwen FisherCorporate Communications Amarin Corporation plcIn U.S.: +1 (908) 325-0735 pr@amarincorp.com

References

1 Bhatt DL, Steg PG, Miller M, et al. Cardiovascular Risk Reduction with Icosapent Ethyl for Hypertriglyceridemia. N Engl J Med 2019; 380:11-22.2 Bhatt DL, Miller M, Brinton EA, et al. REDUCE-IT USA: Results from the 3,146 Patients Randomized in the United States. Circulation 2019. DOI: 10.1161/CIRCULATIONAHA.119.044440.3 Bhatt DL, Steg PG, Miller M, et al. Effects of Icosapent Ethyl on Total Ischemic Events: From REDUCE-IT. J Am Coll Cardio. 2019; 73:2791-2802.4 American Heart Association. 2018. Disease and Stroke Statistics-2018 Update.5 American Heart Association. 2017. Cardiovascular Disease: A Costly Burden for America Projections Through 2035.6 Ganda OP, Bhatt DL, Mason RP, et al. Unmet need for adjunctive dyslipidemia therapy in hypertriglyceridemia management. J Am Coll Cardiol. 2018;72(3):330-343.7 Budoff M, Triglycerides and triglyceride-rich lipoproteins in the causal pathway of cardiovascular disease. Am J Cardiol. 2016;118:138-145.8 Toth PP, Granowitz C, Hull M, et al. High triglycerides are associated with increased cardiovascular events, medical costs, and resource use: A real-world administrative claims analysis of statin-treated patients with high residual cardiovascular risk. J Am Heart Assoc. 2018;7(15):e008740.9 Nordestgaard BG, Triglyceride-rich lipoproteins and atherosclerotic cardiovascular disease - New insights from epidemiology, genetics, and biology. Circ Res. 2016;118:547-563.10 Nordestgaard BG, Varbo A. Triglycerides and cardiovascular disease. Lancet. 2014;384:626635.

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REDUCE-IT USA Results, in Prespecified Subgroup Analyses of Landmark REDUCE-IT Global Study, Showed Robust Cardiovascular Risk Reductions Across a...

Recommendation and review posted by Bethany Smith

Eight years of work and some well-timed coincidences led Donald McDonnell and his lab to a discovery that could change the lives of breast cancer patients.

McDonnell, chair of the department of pharmacology and cancer biology, researches a type of breast cancer called estrogen-receptor positive cancera type in which tumor cells grow in response to the hormone estrogen. He said ER-positive breast cancer makes up about 75% of cases of the disease.

Doctors can treat this kind of cancer with endocrine therapy, which uses anti-estrogen hormones to stop tumors from growing, according to the Mayo Clinic. However, McDonnell said that some cancers are resistant to this treatment, which leaves traditional chemotherapy as the only option.

The product of eight years of research conducted in McDonnells lab, recently published in Cell Reports, could change that. Researchers discovered a way to stop tumors from growing by using antibodies to target two proteins found in endocrine-therapy-resistant cancer cells.

This would likely be another relatively benign, in terms of side effects, therapy, so that it potentially could extend the life of patients who have late-stage disease, McDonnell said.

Instead of trying to prevent cells from becoming resistant, the researchers in McDonnells lab explored ways to exploit the differences between resistant and non-resistant cells, creating treatments that would be uniquely effective in cells that do not respond to traditional therapy.

Researchers identified two proteins, AGR2 and LYPD3, present in therapy-resistant cancers. The proteins interact with each other, and the researchers thought that they might be able to prevent tumor growth by targeting them with a drug.

McDonnell said that he was presenting these results at a Duke Cancer Institute research meeting when Jim Abbruzzese, D.C.I. professor of medical oncology at Duke, told him about research that identified LYPD3 in pancreatic cancer cells. Moreover, Abbruzzese said that a researcher at the MD Anderson Cancer Center had developed antibodies that neutralized the protein.

Interested, McDonnell went to Texas.

Charles Logsdon, professor and chair of cancer biology at MD Anderson, had created antibodies to both proteins and licensed them to a company called Viba Therapeutics. Viba, a company in which McDonnell owns stock, provided the antibodies to researchers in McDonnells lab, who injected them into mice with endocrine-resistant breast cancer.

The results were striking: The tumors stopped growing.

I think a lot of basic science is kind of putting pieces together until you make that connection, said Kimberly Darlington, a former M.D.-Ph.D. student and researcher in McDonnells lab, of the discoveries that led to the teams breakthrough.

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McDonnells team submitted their findings for publication, he said, but the reviewers wanted more from them. Animal models were one thing, but were the proteins found in human breast cancer as well?

Answering that question took more collaboration. McDonnell turned to Mitch Dowsett, a professor of biochemical endocrinology at the Royal Marsden Hospital in London who oversees large collections of data on tumors in humans.

McDonnells team worked with tissue samples that Dowsett had gathered. Their findings matched what they had seen so far: AGR2 and LYPD3 were highly prevalent in cells from tumors that were resistant to traditional therapy.

My goal in starting this project was to understand the process and trials and tribulations of drug discovery, and I feel like weve sort of gone through the gamut, Darlington said, from working with cells to performing mouse studies to finding evidence that their treatment could work in humans.

Today, McDonnell runs a lab on the second floor of Dukes Levine Science Research Center that manages to be both spacious and, in some places, cluttered. Researchers peer into microscopes, machinery covers the countertops and shelves feature an array of bottles and boxes.

This is not where McDonnell thought he would end up, he said. He grew up in Limerick, Ireland, and he earned a scholarship to study marine biology at the National University of Ireland, Galway. That plan changed when he met Mary Downes at a community dance, whose mother had been diagnosed with breast cancer earlier that day.

McDonnell and Downes started dating, and Downes mother would have him go to the library to research her disease. That research inspired McDonnell to go to the group that had given him the scholarship and ask to use the money to study breast cancer.

The guy who was the head of the scholarship committee said, You know, my daughter or my wife may get breast cancer some day. Take your scholarship and go and study breast cancer, McDonnell said.

McDonnell studied metabolism and immunology at NUI Galway, graduating in 1983. By then, he and Downes had married and the couple moved to Houston so that McDonnell could pursue a Ph.D. at Baylor.

McDonnell is now 58, and he said he has had an amazing career in breast cancer research.

I would have been cold, poor and wet for the rest of my life, as a marine biologist on the west coast of Ireland, he said, laughing.

Today, McDonnells lab is working with Viba to develop versions of their antibodies that work in humans. McDonnell said that he is hopeful about the results, and a drug could be on the market soon.

Originally posted here:
Years of work lead Duke researcher to clues behind treating breast cancer - Duke Chronicle

Recommendation and review posted by Bethany Smith

Q: How common is breast cancer in men?

A: Men do get breast cancer, but at much lower numbers than women.

About 2,200 American men are diagnosed with breast cancer each year, compared with about 245,000 women, according to the Centers for Disease Control and Prevention. About 460 men die from the disease annually, compared to 41,000 women who die.

The risk of a man developing breast cancer is low, but men are more likely to die from breast cancer than their female counterparts, according to a Vanderbilt University study recently published in JAMA Oncology.

Breast cancer isnt just a disease for females, Dr. Xiao-ou Shu, senior author of the Vanderbilt study, told the online publication STAT. Men diagnosed with breast cancer have a worse survival rate, and we dont understand the exact reason for the disparity.

Men, just like women, are encouraged to do self-breast exams. The National Comprehensive Cancer Network recommends men who are 35 or older and at higher risk for breast cancer have an annual clinical breast exam.

Mathew Knowles, father and former manager of Beyonc and Solange, put male breast cancer in the spotlight by speaking publicly about his diagnosis. A mammogram showed he had stage 1A breast cancer, and he had a mastectomy in July, according to the New York Times.

Testing revealed that Knowles had the BRCA2 gene mutation, an inheritable trait that increases the risk of some cancers.

Knowles found his cancer at an early stage, but most men are diagnosed when the cancer is more advanced, said Dr. Halle Moore, an oncologist at the Cleveland Clinic.

People might have a problem and dont come forward, Moore said. Theres not as much awareness as there could be.

Men with breast cancer often feel isolated and invisible, said Betsy Kohn, chief program officer for The Gathering Place, which offers free programs to help people and families coping with cancer.

When they tell others about their diagnosis, they are often met with disbelief. Doctor offices decorated in pink send a message that men are interlopers in an all-female space.

They dont have a lot of people to talk to, Kohn said. She counsels men with breast cancer to find a support group where they can share feelings, and be willing to ask for help.

Men with breast cancer have similar treatment options as women, including surgery, radiation, chemotherapy and hormone therapy. Targeted therapy, which are drugs that target cell changes in tumors, are also an option.

Here is more information about breast cancer in men, from the American Cancer Society.

Risk factors:

Symptoms:

Women have similar breast cancer symptoms.

Resources for more information:

American Cancer Society in Ohio

A Gathering Place

Male Breast Cancer Coalition

HIS Breast Cancer Awareness

If you are a man who has had breast cancer, contact patient advocacy writer Julie Washington for a future story. Include your name and city where you live; your comments may be published.

In her Health Matters column, Washington will answer readers questions about navigating health-care systems. (She will not address individual treatments.) Your comments may be published in a future story. Send questions and comments for publication including your name, city and daytime phone number to jwashington@plaind.com. You can also find Julie on Twitter @JulieEWash.

Link:
Mens risk of breast cancer is low, but mortality is high: Health Matters - cleveland.com

Recommendation and review posted by Bethany Smith

The worldwide market for Thyroid Hormone Disorder Drug is expected to grow at a CAGR of roughly over the next five years, will reach million US$ in 2024, from million US$ in 2019.

Access Report Details at: https://www.themarketreports.com/report/global-thyroid-hormone-disorder-drug-market-by-manufacturers-regions-type-and-application-forecast

Market share of global Thyroid Hormone Disorder Drug industry is dominate by companies like Novo Nordisk, Sanofi, Merck, Eli Lilly, AstraZeneca, AbbVie and others which are profiled in this report as well in terms of Sales, Price, Revenue, Gross Margin and Market Share (2017-2018).

With the help of 15 chapters spread over 100 pages this report describe Thyroid Hormone Disorder Drug Introduction, product scope, market overview, market opportunities, market risk, and market driving force. Later it provide top manufacturers sales, revenue, and price of Thyroid Hormone Disorder Drug, in 2017 and 2018 followed by regional and country wise analysis of sales, revenue and market share. Added to above, the important forecasting information by regions, type and application, with sales and revenue from 2019 to 2024 is provided in this research report. At last information about Thyroid Hormone Disorder Drug sales channel, distributors, traders, dealers, and research findings completes the global Thyroid Hormone Disorder Drug market research report.

Market Segment by Regions, regional analysis covers:

North America (USA, Canada and Mexico)

Europe (Germany, France, UK, Russia and Italy)

Asia-Pacific (China, Japan, Korea, India and Southeast Asia)

South America (Brazil, Argentina, Columbia, etc.)

Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa)

Market Segment by Type, covers:

Injection

Oral

Others

Market Segment by Applications, can be divided into

Hospital

Clinic

Medical Center

Others

Purchase this premium research report at: https://www.themarketreports.com/report/buy-now/1488400

Table of Contents

1 Market Overview

2 Manufacturers Profiles

3 Global Thyroid Hormone Disorder Drug Market Competitions, by Manufacturer

4 Global Thyroid Hormone Disorder Drug Market Analysis by Regions

5 North America Thyroid Hormone Disorder Drug by Countries

6 Europe Thyroid Hormone Disorder Drug by Countries

7 Asia-Pacific Thyroid Hormone Disorder Drug by Countries

8 South America Thyroid Hormone Disorder Drug by Countries

9 Middle East and Africa Thyroid Hormone Disorder Drug by Countries

10 Global Thyroid Hormone Disorder Drug Market Segment by Type

11 Global Thyroid Hormone Disorder Drug Market Segment by Application

12 Thyroid Hormone Disorder Drug Market Forecast (2019-2024)

13 Sales Channel, Distributors, Traders and Dealers

14 Research Findings and Conclusion

15 Appendix

Ask your report related queries at: https://www.themarketreports.com/report/ask-your-query/1488400

Continued here:
Global Thyroid Hormone Disorder Drug Industry: Sales, Revenue, Market Share and Competition by Manufacturer Covered in a Latest Research - Market...

Recommendation and review posted by Bethany Smith

This roundup first appeared in our weekly Wednesday newsletter. If youd like to get our sector-special roundups straight to your inbox each week sign up here!

On-demand video consultations with doctors have proven popular healthtech startup apps like Kry and Babylon have been downloaded more than 1.2m times in Europe as patients show that theyre keen for flexible checkups.

Now it turns out that people want on-demand video consultations for their pets, too.

FirstVet is a three-year-old Swedish startup that puts pet owners in touch with vets via an app. Its expanded into neighbouring Norway, Denmark and Finland, as well as the UK and has clocked up more than 200,000 registered users.

One Trustpilot reviewer said its great for emergencies that probably arent emergencies. Other reviewers were pleased that FirstVet saved them a trip to a clinic and a hefty bill.

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Like many doctor apps FirstVet has built partnerships with several insurance companies, including Bought By Many in the UK, making the service free for some users. For other UK customers it costs 20-30 per consultation.

Dogs are the most commonly treated pet, followed by cats. Horses account for 4% of consultations, while perhaps the most unusual treatment FirstVet has assisted with was organising an x-ray for a goldfish.

With its latest injection of funding an 18.5m Series B led by London-based fund OMERS Ventures, with participation from Creandum FirstVet plans to launch in other markets, such as France, Germany and the US. Theres big money to be made heading to North America: the US veterinary care market was worth $70bn in 2017, compared with only 9bn in Germany.

FirstVet currently has a team of 35 and works with 150 vets.

Pharma giant Bayers venture capital arm, Leaps by Bayer, led a $25m round of investment into UK medtech startup Medopad.

Its not the only big firm keen on the company; Medopad also works with Johnson & Johnson, GlaxoSmithKline (GSK) and Chinese internet giant Tencent, amongst others.

Medopad, founded in London in 2011, builds and tracks digital biomarkers (i.e. indicators of illness or disease picked up by apps and wearables). These help doctors monitor patients conditions via an app.

Its also integrated several other healthtech companies within its platform, such as medical equipment firm Medtronic and heart rhythm monitor FibriCheck. This means clinicians (and patients) can use Medopad to track several conditions rather than using a separate app for each.

Many healthtech startups are making a platform play: from Berlin-based digital diagnosis app Ada to Paris-based health insurance provider Alan all aspire to be a one-stop-shop for patients health needs.

Few European healthtech businesses have landed as many corporate and hospital partnerships as Medopad, however. Earlier this year, it began a clinical trial with Tencent to test the use of artificial intelligenceto diagnose patients with Parkinsons disease. It also signed a three-year contract with one UK hospital, The Royal Wolverhampton NHS Trust, to offer remote support to patients living with heart problems and diabetes. Medopad has also begun acquiring competitors; last September it bought US rival Sherbit.

Inne is far from the only fertility and contraception startup in Europe. There are now quite a few, including Natural Cycles, a birth control app, Moody Month, an app which helps women track their hormones and Clue, which helps women track their periods. Many of these aim to help women return to their natural cycle and move away from hormone-control pills.

Berlin-based Inne has developed a saliva biosensor device to help women work out when theyll be most fertile. The product, which can be used to get pregnant or avoid getting pregnant, will launch later this year.

London venture capital firm Blossom Capital led the round, with prominent angel investors Taavet Hinrikus and Tom Stafford also participating.

Inne joins the booming world of European femtech from digital doctors specifically catering to womento menopause tech.

See the original post:
FirstVet raises 18.5m and other European healthtech news - Sifted

Recommendation and review posted by Bethany Smith

Teens and young adults with anorexia are at risk of life-threatening illness even if their weight falls within a "normal" range.

That's the conclusion of a new study that looked at "atypical anorexia," or cases in which patients show all the symptoms of anorexia with the exception of low body weight. In the past, these cases were considered less severe than typical anorexia cases, but the new study found that both types show the same signs of severe malnutrition. "Patients with atypical anorexia are just as sick, medically but they may be even sicker, psychologically," said Dr. Neville Golden, a professor of pediatrics at the Stanford School of Medicine and co-author of the new study. Although recognized in the diagnostic manual for mental health disorders, the DSM-5, atypical anorexia may remain underdiagnosed, Golden said.

"The assumption is that doctors in the community are not recognizing it," he said. The oversight may place patients at risk of cardiac arrest, bone degradation and even death, Golden and his colleagues found.

The new study, published Nov. 5 in the journal Pediatrics, shows that there's no connection between an anorexic patient's weight and the actual severity of their condition. In the end, the number on the scale matters far less than the sheer amount of weight patients lose over the course of their illness both normal-weight and underweight patients fare worse the more weight they drop.

Related: Understanding Weight: BMI & Body Fat

"There's no weight or BMI that equals [an] eating disorder," said Dr. Casey Cottrill, the medical director of the eating disorders program at Nationwide Children's Hospital in Columbus, Ohio, who was not involved with the study. Over the last five to 10 years, the number of normal-weight people hospitalized for anorexia treatment has spiked, she said. Recent studies estimate that one-third of patients admitted to hospitals for anorexia treatment are of normal weight. In both atypical and typical cases, the signs of malnutrition appear the same, but patients of normal or above-average weight may suffer longer before being noticed.

In light of this, doctors must watch for signs of disordered eating and malnutrition in all patients, regardless of size, Cottrill said.

Although atypical anorexia has gained recognition, still, "when one thinks of malnutrition, one thinks of low weight," Golden said. To learn whether low-weight anorexic patients actually fare worse in clinic, Golden and his colleagues organized the largest, most comprehensive assessment of normal-weight adolescents with anorexia to date.

The study compared 50 teens and young adults with atypical anorexia with 66 patients who met the traditional diagnostic criteria, meaning their weight fell below 85% of what would be expected for their height and age. The participants ranged in age from 12 to 24 years old and received treatment for their disorders during the study. More than 90% of participants were female. (Anorexia is about 3 times more common in females compared with males, according to the National Eating Disorders Association (NEDA).)

The authors compared the patients' current weights, histories of weight loss and vital signs; and found that regardless of participants' weight at admission, those with more dramatic weight losses appeared more severely ill.

Patients who lost a large amount of weight, fast, displayed the lowest heart rates among those in the study. In fact, for every 2% increase in the rate of weight loss per month, patients' heart rates measured 1 beat per minute slower in the hospital. A dangerously low heart rate points to a larger problem: poor nutrition leaves the heart with too little fuel to pump properly while also forcing the body to break down heart tissue for much-needed energy, according to NEDA. Clinicians usually hospitalize patients whose heart rates clock below 50 beats per minute, as their condition can quickly deteriorate into complete heart failure, Cottrill said.

Related: What Is Mental Health?

Patients with atypical and typical anorexia showed similar dips in other critical measures of health. Both groups had dangerously low blood pressure and became dizzy when moving from lying down to sitting up or standing. Both groups showed deficiencies in key electrolytes such as potassium, phosphorus and magnesium nutrients that help vital organs like the heart running smoothly. The patients who lost the most weight, or had been losing weight for an extended period, had the lowest electrolyte levels.

All female patients who had begun menstruating stopped having regular periods, meaning their bodies were no longer producing enough estrogen to maintain their normal cycles. Without estrogen, the growing patients' bones could not absorb calcium as they should during puberty, Cottrill said.

Overall, both groups of patients appeared similar on all counts except one. Those with atypical anorexia actually ranked worse on a questionnaire designed to measure the severity of their eating disorder psychopathology. The poor scores suggest that those with atypical anorexia may be more fixated on losing weight, restricting their food intake and burning off calories than those with typical anorexia. Anecdotally, the patients seemed "very fearful of getting back to their [original] weight," Golden said.

Future research should investigate the best treatment for cases of atypical anorexia, particularly cases in which patients are overweight or obese, since very little data exists in this area, Cottrill said. Weight gain normally comes along with both the physical and psychological treatment of anorexia, but with overweight patients, it's difficult to gauge how much weight they need to regain. More research must be done on how different bodies react to malnutrition and how best to treat patients of different sizes, Cottrill said.

In addition, when treating obesity, doctors must learn how to help people lose weight sustainably, without resorting to drastic measures, Golden said. By monitoring patients more closely, perhaps physicians can catch poor habits before they fester into a full-fledged eating disorder, he suggested. The first step, of course, is to raise awareness of what disordered eating looks like.

"I think there's a lack of awareness of atypical anorexia nervosa, even among clinicians," said Dr. Avinash Boddapati, a child and adolescent psychiatrist in the Northwell Health network, who was not involved with the study. As a psychiatrist, Boddapati said he can address the underlying emotional distress and harmful coping mechanisms that lead to disordered eating. But to tackle the problem head-on, pediatricians and parental guardians need to work together to monitor signs of atypical anorexia.

"The big take home message is to focus, not just on the weight, but on the rate of weight loss," he said.

Psychiatrists can also screen for rapid and extensive weight loss in their patients, "even kids [who fall] within a normal weight range," said Dr. Peng Pang, an adolescent psychiatrist at Staten Island University Hospital in New York. First, mental health professionals should ensure that their patients are physically stable, and refer them to a hospital if their health may be compromised, said Pang, who wasn't involved with the study. Then, once the patient's vital signs are restored, psychiatrists can work with patients to find new, sustainable coping mechanisms.

"Regardless of the body weight, I think the message is that you have to intervene, immediately and aggressively," Pang said.

Originally published on Live Science.

Read more here:
Teens with Anorexia May Be 'Dangerously Ill' Even if They Are Not Underweight - Livescience.com

Recommendation and review posted by Bethany Smith

Addison disease occurs due to the injury of the adrenal cortex which causes insufficient generation of the hormone aldosterone and cortisol.Indications of Addisons disease are known as primary adrenal insufficiency, resulting from insufficient production of two hormones cortisol and aldosterone.

Access Report Details at: https://www.themarketreports.com/report/global-addison-disease-testing-market-by-manufacturers-regions-type-and-application-forecast

Market share of global Addison Disease Testing industry is dominate by companies like NHS.UK, Mayo Clinic, VCA Animal Hospital, NIDDK, Cleveland Clinic, WebMD, BMJ Best Practice, National Organization for Rare Disorders, Core Diagnostics Private Limited, Laboratory Corporation of America Holdings, Sonora Quest Laboratories and others which are profiled in this report as well in terms of Sales, Price, Revenue, Gross Margin and Market Share (2017-2018).

With the help of 15 chapters spread over 100 pages this report describe Addison Disease Testing Introduction, product scope, market overview, market opportunities, market risk, and market driving force. Later it provide top manufacturers sales, revenue, and price of Addison Disease Testing, in 2017 and 2018 followed by regional and country wise analysis of sales, revenue and market share. Added to above, the important forecasting information by regions, type and application, with sales and revenue from 2019 to 2024 is provided in this research report. At last information about Addison Disease Testing sales channel, distributors, traders, dealers, and research findings completes the global Addison Disease Testing market research report.

Market Segment by Regions, regional analysis covers:

North America (USA, Canada and Mexico)

Europe (Germany, France, UK, Russia and Italy)

Asia-Pacific (China, Japan, Korea, India and Southeast Asia)

South America (Brazil, Argentina, Columbia, etc.)

Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa)

Market Segment by Type, covers:

Laboratory testing

Imaging studies

Market Segment by Applications, can be divided into

Clinics

Hospitals

Diagnostics laboratories

Purchase this premium research report at: https://www.themarketreports.com/report/buy-now/1499860

Table of Contents

1 Market Overview

2 Manufacturers Profiles

3 Global Addison Disease Testing Market Competitions, by Manufacturer

4 Global Addison Disease Testing Market Analysis by Regions

5 North America Addison Disease Testing by Countries

6 Europe Addison Disease Testing by Countries

7 Asia-Pacific Addison Disease Testing by Countries

8 South America Addison Disease Testing by Countries

9 Middle East and Africa Addison Disease Testing by Countries

10 Global Addison Disease Testing Market Segment by Type

11 Global Addison Disease Testing Market Segment by Application

12 Addison Disease Testing Market Forecast (2019-2024)

13 Sales Channel, Distributors, Traders and Dealers

14 Research Findings and Conclusion

15 Appendix

Ask your report related queries at: https://www.themarketreports.com/report/ask-your-query/1499860

More:
Global Addison Disease Testing Market Forecast to 2024 by Types, Application and by Regions - Market Research Newspaper

Recommendation and review posted by Bethany Smith

LOS ANGELES: Wellness brand HUM Nutrition, the leader in the beauty vitamin and supplement category, launches its latest revolutionary product, MIGHTY NIGHT, to boost skin cell renewal during sleep.

Mighty Night uses clean, clinically proven ingredients in effective dosages that work while you sleep resulting in a fresh complexion each morning. The proprietary formula boosts overnight renewal by supporting skin cell turnover, scavenging free radicals, promoting optimal sleep and improving skin texture.

During sleep, the body produces more collagen and melatonin both known to reduce fine lines and wrinkles. Levels of the stress hormone cortisol fall during sleep, which helps skin to repair daytime damage. And, the human growth hormone, responsible for accelerating skin's repair and cell regeneration, is released during sleep.

Mighty Night ingredients include Ubiquinol, the most absorbable form of CoQ10 which protects the skin cell's membrane and supports overall renewal; Ceramides to lock in moisture and boost elasticity; Ferulic Acid proven to scavenge free radicals; and, a clinically studied combination of Valerian Root, Hops and Passion Flower that helps to promote optimal sleep.

Sleep is when your skin repairs itself, grows new cells and fortifies against moisture loss and free radical damage. Valerian Root and Hops are two herbs I recommend for better sleep quality, which is critical for overnight recovery, says Dr. Breus PHD, aka The Sleep Doctor.

Dermatologist Dr. Julie Russak of the Russak Dermatology Clinic in New York says Ferulic acid and ceramides offer skin benefits while you sleep by improving the protective barrier of skin and strengthening it. When our skin barrier is at its optimal state, we appear healthier. HUM's Mighty Night is a responsibly sourced, multi-beneficial supplement I trust and recommend to my patients.

Mighty Night is available at http://www.humnutrition.com and http://www.sephora.com beginning October 18th. It retails for $40 for a 30-day supply (60 capsules) and is vegan, vegetarian, Non-GMO, gluten-free and sustainably sourced.

Take 2 at bedtime and expect results in 4 to 6 weeks.Here's to a peaceful beauty rest, Sleeping Beauty.

Read more here:
HUM Nutrition Launches Mighty Night to optimize beauty sleep from within, United States News#244481 - New Kerala

Recommendation and review posted by Bethany Smith

Inovio Pharmaceuticals breached a contract to supply needle-free injectors after learning they would be used to inject testosterone by transgender men, according to a lawsuit filed Nov. 1 in San Diego Superior Court.

BLS Pharma says Inovio was cooperative until it learned the biomedical companys ZetaJet injectors would be used by the transgender men, instead of just men with insufficient testosterone, a condition called male hypogonadism. BLS asked Inovio for its reasons, and the company failed to give a valid reply, according to the lawsuit.

Inovio rejects the claim. The lawsuit has absolutely no merit, said Jeff Richardson, an Inovio spokesman.

San Diego is where Inovio performs research, development, engineering and manufacturing. Company headquarters is in Plymouth Meeting, Pennsylvania. BLS Pharma is headquartered in Lake Forest, Calif.

The civil lawsuit says the monetary loss totals $72.9 million under a conservative estimate. Besides breach of contract, the lawsuit also alleges fraud and negligent misrepresentation.

Inovio acquired ZetaJet in 2016 when it paid $4.3 million in stock and $1.2 million cash for BioJect, its maker. The device uses high pressure supplied by a spring to force liquid medicine through the skin.

The lawsuit says that sale included a provision to sell ZetaJets to the sellers upon request, negotiating in good faith to supply the devices at a 15 percent premium to production cost.

The sellers formed BLS to continue work already planned to enter the needle-free injection market, including testosterone, the lawsuit said. The combination of device and needle-free testosterone syringe would allow patients to inject themselves with greater ease and comfort than by using a needle.

Both men with hypogonadism and biological women becoming transgender men require continual testosterone supplementation to maintain normal male levels of the hormone. That market is valued at $1.3 and $1.8 billion annually, the lawsuit said.

In early 2017, BLS told Inovio it planned to buy the ZetaJets under the contract, for use in a planned clinical trial. It asked for 25,000 of them in April, and negotiations proceeded over the summer while BLS discussed its plans with the FDA, which regulates clinical trials.

On Sept. 7, BLS told Inovio that it had received favorable news about testing the system for transgender men in addition to men with insufficient testosterone.

The following Monday, September 11, 2017, Defendants requested a call with Plaintiff, the lawsuit stated.

At that time Defendants advised Plaintiff that they were rejecting the syringe order, would not authorize BLS to take over manufacturing of the syringes, that their legal department would be sending a letter terminating the relationship, and that they would return the check previously issued by BLS for payment.

According to the lawsuit, Inovio gave as its reason, We dont need transgender, we have Gates.

BLS said it interpreted that reply as meaning that Inovio was already supporting a different publicizable public interest effort through a separate project with the Gates Foundation.

In the following months, BLS said, it tried and failed to find an alternative supplier of the injectors, leaving it without any way to test its product.

The lawsuit also names as defendant Genetronics, a San Diego-based Inovio predecessor. Genetronics purchased Norway-based Inovio in 2005, and shortly thereafter changed the combined companys name to Inovio. The lawsuit says Genetronics is now an Inovio subsidiary.

A civil case management conference has been set for June 19, 2020, according to a Superior Court filing for the case. The case number is 37-2019-00058344-CU-BC-CTL.

Read this article:
Lawsuit claims contract breached over transgender testosterone therapy - The San Diego Union-Tribune

Recommendation and review posted by Bethany Smith

Another seminar is again advertised in The Union. I first thought it might help my wife with neuropathy until I did some extensive research.

First off, Medicare does not cover stem cell injections. Bone marrow stem cell injections range from $2,000 to $5,000 or more. Read Consumer Research report at: https://www.consumerreports.org/medical-treatments-procedures/trouble-with-stem-cell-therapy.

Stem cell treatments are widely accepted only for two broad medical indications: to help treat a handful of blood disorders including leukemia and some forms of anemia and in some cases to help burn victims. Ask questions. Any doctor who offers stem cell therapy should be able to explain where the cells will come from, what will be done to them before theyre injected into your body, and how, exactly, they will resolve your illness or injury. He or she should also be able to offer you proof of safety and efficacy, even for experimental treatments. Dont rely on patient testimonials.

Stem cells survive much longer than ordinary cells, increasing the chance that they might accumulate genetic mutations. It might take only a few mutations for one cell to lose control over its self-renewal and growth and become the source of cancer. Please do your own research.

Gary Pesselt

Grass Valley

See more here:
Gary Pesselt: Vitality Healthcare is it worth the cost? - The Union of Grass Valley

Recommendation and review posted by Bethany Smith

Myelofibrosis or osteomyelofibrosis is a myeloproliferative disorder which is characterized by proliferation of abnormal clone of hematopoietic stem cells. Myelofibrosis is a rare type of chronic leukemia which affects the blood forming function of the bone marrow tissue. National Institute of Health (NIH) has listed it as a rare disease as the prevalence of myelofibrosis in UK is as low as 0.5 cases per 100,000 population. The cause of myelofibrosis is the genetic mutation in bone marrow stem cells. The disorder is found to occur mainly in the people of age 50 or more and shows no symptoms at an early stage. The common symptoms associated with myelofibrosis include weakness, fatigue, anemia, splenomegaly (spleen enlargement) and gout. However, the disease progresses very slowly and 10% of the patients eventually develop acute myeloid leukemia. Treatment options for myelofibrosis are mainly to prevent the complications associated with low blood count and splenomegaly.

To Remain Ahead Of Your Competitors, Request for a Sample Here @https://www.persistencemarketresearch.com/samples/11341

The global market for myelofibrosis treatment is expected to grow moderately due to low incidence of a disease. However, increasing incidence of genetic disorders, lifestyle up-gradation and rise in smoking population are the factors which can boost the growth of global myelofibrosis treatment market. The high cost of therapy will the growth of global myelofibrosis treatment market.

The global market for myelofibrosis treatment is segmented on basis of treatment type, end user and geography:

As myelofibrosis is considered as non-curable disease treatment options mainly depend on visible symptoms of a disease. Primary stages of the myelofibrosis are treated with supportive therapies such as chemotherapy and radiation therapy. However, there are serious unmet needs in myelofibrosis treatment market due to lack of disease modifying agents. Approval of JAK1/JAK2 inhibitor Ruxolitinib in 2011 is considered as a breakthrough in myelofibrosis treatment. Stem cell transplantation for the treatment of myelofibrosis also holds tremendous potential for market growth but high cost of therapy is foreseen to limits the growth of the segment.

On the basis of treatment type, the global myelofibrosis treatment market has been segmented into blood transfusion, chemotherapy, androgen therapy and stem cell or bone marrow transplantation. Chemotherapy segment is expected to contribute major share due to easy availability of chemotherapeutic agents. Ruxolitinib is the only chemotherapeutic agent approved by the USFDA specifically for the treatment of myelofibrosis, which will drive the global myelofibrosis treatment market over the forecast period.

Geographically, global myelofibrosis treatment market is segmented into five regions viz. North America, Latin America, Europe, Asia Pacific and Middle East & Africa. Northe America is anticipated to lead the global myelofibrosis treatment market due to comparatively high prevalence of the disease in the region.

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Some of the key market players in the global myelofibrosis treatment market are Incyte Corporation, Novartis AG, Celgene Corporation, Mylan Pharmaceuticals Ulc., Bristol-Myers Squibb Company, Eli Lilly and Company, Taro Pharmaceuticals Inc., AllCells LLC, Lonza Group Ltd., ATCC Inc. and others.

See more here:
Myelofibrosis Treatment Market To Witness an Outstanding Growth During 2016-2022 - Zebvo

Recommendation and review posted by Bethany Smith

Whoever saves a single life is considered by the Talmud to have saved the whole world.

In August 2013, Jeffrey Altadonna, who was on a Birthright trip, was tested at a bone marrow testing drive at the Jerusalem Gate Hotel.

It was perfectly ordinary summer day when the 29-year-old accountant from Sherman Oaks, California received the fateful phone call.

A 77-year-old woman from Los Angeles was the perfect stranger that he was deemed to save.

Diane Gebel, a widow from Cyprus, California was diagnosed with Acute Myeloid Leukemia (AML). Her husband had passed away right before she was diagnosed with cancer.

For an entire year, the donor and recipient need to remain anonymous to each other, but last week, the time came for the two to finally meet.

The two were honored at the Los Angeles One Huge Night Gala event hosted by Gift of Life.

In a statement, Birthright Israel explained that the gala also celebrated the successful 15-year partnership of Birthright Israel and Gift of Life.

This partnership has so far resulted in 83,000 Birthright donors joining the registry, with 1,900 matches made between patients and Birthright donors, and 241 life-saving transplants to date.

Prior to the meeting, Altadonna recalled his Birthright trip explaining that it was really great to see that part of the world, to go to where its our given right to visit.

It had a profound effect on my friendships and cultural Jewish identity to see that Jewish people are one people, and we have each others backs, he said. It left me with the feeling that I had backing in anything that I wanted to do in my life. It really felt like a family.

He recalled that after being swabbed at the drive, he didnt really think too much of it because everyone did it.

I got the donation call 15 months ago, he explained, adding that he immediately decided to donate. I find it very bizarre, that everyone is telling me Its such a great thing that you are doing. For me, it wasnt an option to say yes or no, its just, Okay, lets do this, Im a match. Im surprised that more people dont donate.

After doing preliminary tests, he took the plunge and donated.

I had to do it early in the morning, it lasted 6-8 hours a marathon blood donation and it was finished,Altadonna continued. It didnt seem all that hard to me.

He made it clear that this opportunity to help only came about because of the Birthright Israel and Gift of Life collaboration.

It wasnt a mission of mine. I wouldnt have gone out of my way to get swabbed, so it only happened as a result of their collaboration, he said.

In an emotional meeting, the two finally met. Of the meeting, Gebel stressed that she is here because of my selfless and generous donor.

For me, it was easy, I just had an infusion, but for him it was hard, she said. It takes a very special person to do that, to actually give the gift of life.

Gebel said she had been waiting to meet him.

I didnt know he was such a good looking guy, Gebel joked. Im here because of him. I was not ready to die. I had too much to live for.

She stressed that she has changed because of my new life.

I take risks, I live my life fully, she explained. My motto has always been that I want to go through life with a Cosmopolitan in one hand and a travel book in the other. Because of my donor, I can do that, so I thank him from the bottom of my heart.

Altadonna called on others to also take the plunge by getting tested and donating.

I stand here for my recipients valor, for her victory. I ask you to do the same: sit and swab today, so someone can swim and live tomorrow, he said.

Birthrights International CEO Gidi Mark said he was proud of our participants who register as donors and the powerful impact of such a simple choice when they get the call that they are the perfect match for a perfect stranger.

It is a great honor that Birthright Israel is able to assist in this mission, he added.

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Perfect match: How Birthright alumni saved the lives of 100s of strangers - The Jerusalem Post

Recommendation and review posted by Bethany Smith

Proud auntie Paola Mayfield says her niece, who has Down syndrome, is in remission from the leukemia that she battled for two years.

Last year I went to Colombia when I was pregnant, I needed to see my sister and my niece, Mayfield wrote alongside a photo of niece standing on a hospital bed with her arm attached to some medical devices. My niece has Down Syndrome and had been battling leukemia for about 2 years. But TODAY we received the greatest news! She is finally cancer free!

Paola said that the journey has been extremely difficult. With tears in my eyes I feel full of joy and happiness because I know how hard it has been the past few years for my sister.

And she wanted others who are struggling and fighting for their lives to know that there is always hope: For those who battle everyday to have another day of life, stay strong and dont lose faith. Thank you [hearts].

Leukemia is the most common type of childhood cancer. The most frequent type of childhood leukemia is acute lymphoblastic leukemia (ALL). Three out every four cases of childhood leukemia are diagnosed as acute, meaning that the leukemia can progress quickly, and if not treated, would probably be fatal within a few months.

Acute lymphoblastic leukemia is a rare cancer thatoccurs when the bone marrow makes too much of a type of white blood cell calledlymphocytes, according to the National Cancer Institute. Signs of childhood ALL include fever and bruising. The disease can be detected using tests that examine the blood and bone marrow. Over time, there has been a lot of improvement in treatments for childhood leukemia.

There are several different approaches to treating the disease, and the treatment plan will depend on the type of ALL. Chemotherapy, radiation, chemotherapy with a stem cell transplant, and targeted therapy are all considered standard treatment, according to the American Cancer Society.

The next most common type of childhood leukemia is called acute myeloid leukemia, which occurs when the bone marrow makes a large number of abnormal blood cells called myeloblasts. As these cells build up, they prevent the growth ofhealthy white blood cells, red blood cells, and platelets.

Learn more about SurvivorNet's rigorous medical review process.

Proud auntie Paola Mayfield says her niece, who has Down syndrome, is in remission from the leukemia that she battled for two years.

Last year I went to Colombia when I was pregnant, I needed to see my sister and my niece, Mayfield wrote alongside a photo of niece standing on a hospital bed with her arm attached to some medical devices. My niece has Down Syndrome and had been battling leukemia for about 2 years. But TODAY we received the greatest news! She is finally cancer free!

Paola said that the journey has been extremely difficult. With tears in my eyes I feel full of joy and happiness because I know how hard it has been the past few years for my sister.

And she wanted others who are struggling and fighting for their lives to know that there is always hope: For those who battle everyday to have another day of life, stay strong and dont lose faith. Thank you [hearts].

Leukemia is the most common type of childhood cancer. The most frequent type of childhood leukemia is acute lymphoblastic leukemia (ALL). Three out every four cases of childhood leukemia are diagnosed as acute, meaning that the leukemia can progress quickly, and if not treated, would probably be fatal within a few months.

Acute lymphoblastic leukemia is a rare cancer thatoccurs when the bone marrow makes too much of a type of white blood cell calledlymphocytes, according to the National Cancer Institute. Signs of childhood ALL include fever and bruising. The disease can be detected using tests that examine the blood and bone marrow. Over time, there has been a lot of improvement in treatments for childhood leukemia.

There are several different approaches to treating the disease, and the treatment plan will depend on the type of ALL. Chemotherapy, radiation, chemotherapy with a stem cell transplant, and targeted therapy are all considered standard treatment, according to the American Cancer Society.

The next most common type of childhood leukemia is called acute myeloid leukemia, which occurs when the bone marrow makes a large number of abnormal blood cells called myeloblasts. As these cells build up, they prevent the growth ofhealthy white blood cells, red blood cells, and platelets.

Learn more about SurvivorNet's rigorous medical review process.

Read the original post:
Tears And Joy This Toddler with Down Syndrome Who Was Battling Leukemia Is Finally Cancer Free - SurvivorNet

Recommendation and review posted by Bethany Smith

According to results of a study published in Blood, children and young adults with relapsed/refractory B-cell acute lymphoblastic leukemia (ALL) who had minimal residual disease (MRD) prior to treatment and received high-dose preconditioning chemotherapy were most likely to respond to a second-generation CD-19 chimeric antigen receptor-T cell (CAR-T) therapy.1

Although it has been estimated that 90% or more of pediatric patients witha diagnosis of ALL will respond to multi-agent chemotherapy, the prognosis forthose with relapsed/refractory disease remains poor. One CD19-directed CAR-Ttherapy, tisagenlecleucel, is approved by the US Food and Drug Administration inpatients up to age 25 years with B-cell precursor ALL who either have refractorydisease or have experienced a second or later relapse.2

This open label, nonrandomized, phase 1 study (Clinical Trial Identifier: NCT01860937), evaluated the toxicity, feasibility, and response of 19-28z CAR-T therapy, a second-generation CD19-directed CAR-T therapy involving T cells expressing a chimeric receptor composed of an anti-CD19 antibody binding site and intracellular domains from the T-cell coactivating receptors, CD28 and the CD3-zeta chain3 in children and young adults up to 25 years of age with very high-risk ALL.1 Inclusion criteria included at least 2 relapses, early bone marrow relapse following complete response (CR), intermediate/late CR with poor response to re-induction therapy, or those with refractory disease, or ineligibility for allogeneic hematopoietic stem cell transplantation (allo-HSCT) or additional chemotherapy.1

The age range of the 25 patients treated with 19-28z CAR-T therapy onstudy was 1 to 22.5 years, with a median age of 13.5 years. Preconditioningchemotherapy involved high-dose cyclophosphamide (15 patients) and low-dosecyclophosphamide (8 patients), with 3 patients in each subgroup also receivingfludarabine.1

Regarding the feasibility of this approach, the prespecified CAR-Tcell dose was achieved for all patients for whom the 19-28z CAR-T therapyprocedure was undertaken.1

With respect to treatment toxicity,approximately one-third of patients experienced a grade 3/4 adverse event,including cytokine release syndrome (CRS) and neurotoxicity in 16% and 28% ofpatients, respectively. With the exception of 1 patient with grade 4 CRS andneurotoxicity who died following refractory Stenotrophomonas septic shock,these adverse events were reversible.1

Of the 24 patients includedin the response analysis, 75% achieved either a CR or a CR with incompletecount recovery (CRi). In the subsets of patients receiving preconditioningchemotherapy with either high- or low-dose cyclophosphamide, the CR/CRi rateswere 94% and 38%, respectively. Furthermore, treatment response was influencedby disease burden as evidenced by the considerably higher CR/CRi rate inpatients with baseline minimal residual disease (ie, less than 5% bone marrowblasts; 93%) compared with morphological evidence of disease at baseline (5% orhigher bone marrow blasts; 50%).1

The CR/CRi rate for thesubset of patients with pretreatment MRD treated with high-dose cytarabine was100%.1

Consolidation allo-HSCT was performed in 83% (15) of the patientsresponding to CAR-T therapy, with a median time from CAR-T infusion toallo-HSCT of 57 days. At a median follow-up of 28.6 months for respondingpatients, over half of these patients (8) were alive and had no evidence ofdisease.1

In their concluding remarks, the study authorscommented that thisanalysis has allowed us to determine the toxicity profile, confirm feasibility,evaluate response of this approach, and provide a direct comparison of the sameCD19-specific CAR T cell product that was previously published[3] inadult patients for the same indication.

The authors went on to highlight the findingof a reversible toxicity profile in the patients within their study as well asthe impact of preconditioning chemotherapy dose intensity and minimal pretreatmentdisease burden on response.

They further noted that within this cohort,the long-term persistence of response is encouraging, and in our primarilytransplant-naive patient population, the ability to proceed to allo-HSCT hasdemonstrated a favorable overall survival, manageable toxicity, and limitedincidence of relapse.

References

Here is the original post:
19-28z CAR-T Therapy in Children and Young Adults With Relapsed/Refractory ALL: Promising Early Results - Cancer Therapy Advisor

Recommendation and review posted by Bethany Smith